Publications

2019-2015 / 2014-2010 / 2009-2005 / 2004-2001

Kohn DB., et al. (2020) « Lentiviral gene therapy for X-linked chronic granulomatous disease » Nat Med 26(2), 200-206.
Perrin A., et al. (2020) « A new congenital multicore titinopathy associated with fast myosin heavy chain deficiency » Ann Clin Transl Neurol 7(5), 846-854.
Romano O., et al. (2020) « GATA Factor-Mediated Gene Regulation in Human Erythropoiesis » iScience 23(4), 101018.
Ronzitti G., et al. (2020) « Human Immune Responses to Adeno-Associated Virus (AAV) Vectors » Front Immunol 11, 670.

2019

Annoussamy, M., et al. (2019). « X-linked myotubular myopathy: A prospective international natural history study. » Neurology 92(16): e1852-e1867.
Buning, H., et al. (2019). « Breaking the Barriers of Genetic and Metabolic Disorders. » Hum Gene Ther 30(10): 1177-1179.
Colella, P. and F. Mingozzi (2019). « Gene Therapy for Pompe Disease: The Time is now. » Hum Gene Ther 30(10): 1245-1262.
Collaud, F., et al. (2019). « Preclinical Development of an AAV8-hUGT1A1 Vector for the Treatment of Crigler-Najjar Syndrome » Mol Ther Methods Clin Dev 12: 157-174.
Lostal, W., et al. (2019). " « In Vivo Monitoring of Calpain Activity by Forster Resonance Energy Transfer » Methods Mol Biol 1915: 57-66.
Pignani, S., et al. (2019). « Tailoring the CRISPR system to transactivate coagulation gene promoters in normal and mutated contexts. » Biochim Biophys Acta Gene Regul Mech 1862(6): 619-624.
Ronzitti, G., et al. (2019). « Progress and challenges of gene therapy for Pompe disease. » Ann Transl Med 7(13): 287.
Scott, D. W. and F. Mingozzi (2019). « Balancing immunity and tolerance in gene therapy for inherited and acquired diseases. » Cell Immunol 342: 103945.
Avila-Polo, R., et al. (2018). « Loss of Sarcomeric Scaffolding as a Common Baseline Histopathologic Lesion in Titin-Related Myopathies. » J Neuropathol Exp Neurol 77(12): 1101-1114.
Colella, P., et al. (2018). « Emerging Issues in AAV-Mediated In Vivo Gene Therapy. » Mol Ther Methods Clin Dev 8: 87-104.
Denard, J., et al. (2018). « AAV-8 and AAV-9 Vectors Cooperate with Serum Proteins Differently Than AAV-1 and AAV-6. » Mol Ther Methods Clin Dev 10: 291-302.
Henriques, S. F., et al. (2018). « Different outcome of sarcoglycan missense mutation between human and mouse. » PLoS One 13(1): e0191274.
Jonson, P. H., et al. (2018). « Novel mutations in DNAJB6 cause LGMD1D and distal myopathy in French families. » Eur J Neurol 25(5): 790-794.
Kuranda, K., et al. (2018). « Exposure to wild-type AAV drives distinct capsid immunity profiles in humans. » J Clin Invest 128(12): 5267-5279.
Mingozzi, F. (2018). « AAV Immunogenicity: A Matter of Sensitivity. » Mol Ther 26(10): 2335-2336.
Oates, E. C., et al. (2018). « Congenital Titinopathy: Comprehensive characterization and pathogenic insights. » Ann Neurol 83(6): 1105-1124.
Poletti, V. and F. Mavilio (2018). « Interactions between Retroviruses and the Host Cell Genome. » Mol Ther Methods Clin Dev 8: 31-41.
Ronzitti, G. and F. Mingozzi (2018). « Combination Therapy Is the New Gene Therapy? » Mol Ther 26(1): 12-14.
Rouillon, J., et al. (2018). « High urinary ferritin reflects myoglobin iron evacuation in DMD patients. » Neuromuscul Disord 28(7): 564-571.
Urbinati, F., et al. (2018). « Gene Therapy for Sickle Cell Disease: A Lentiviral Vector Comparison Study. » Hum Gene Ther 29(10): 1153-1166.
Weber, L., et al. (2018). « An Optimized Lentiviral Vector Efficiently Corrects the Human Sickle Cell Disease Phenotype. » Mol Ther Methods Clin Dev 10: 268-280.

2017

Boucheham, A., et al. (2017). « IpiRId: Integrative approach for piRNA prediction using genomic and epigenomic data. » PLoS One 12(6): e0179787.
Dalichaouche, I., et al. (2017). « Gamma-sarcoglycan and dystrophin mutation spectrum in an Algerian cohort. » Muscle Nerve 56(1): 129-135.
Hardet, R. and F. Mingozzi (2017). « Oral Tolerance: Another Reason to Eat Your Veggies! » Mol Ther 25(2): 311-313.
Morris, E. C., et al. (2017). « Gene therapy for Wiskott-Aldrich syndrome in a severely affected adult. » Blood 130(11): 1327-1335.
Costopoulos, M., et al. (2016). « ISOLD: A New Highly Sensitive Interleukin Score for Intraocular Lymphoma Diagnosis. » Ophthalmology 123(7): 1626-1628.
El Shafey, N., et al. (2016). « Inhibition of the myostatin/Smad signaling pathway by short decorin-derived peptides » Exp Cell Res 341(2): 187-195.
Galy, A. (2016). « Like Angler Fish, CAARs Lure Their Prey » Mol Ther 24(8): 1339-1341.
Gregoire, S., C. Terrada, et al. (2016). « Treatment of Uveitis by In Situ Administration of Ex Vivo-Activated Polyclonal Regulatory T Cells. » J Immunol 196(5): 2109-2118.
Holic, N. and D. Fenard (2016). « Production of Retrovirus-Based Vectors in Mildly Acidic pH Condition » Methods Mol Biol 1448: 41-48.
Hudry, E., C. Martin, et al. (2016). « Exosome-associated AAV vector as a robust and convenient neuroscience tool. » Gene Ther 23(4): 380-392.
Lawlor, M. W., et al. (2016). « Skeletal Muscle Pathology in X-Linked Myotubular Myopathy: Review With Cross-Species Comparisons » J Neuropathol Exp Neurol 75(2): 102-110.
Merten, O. W., M. Hebben, et al. (2016). « Production of lentiviral vectors. » Mol Ther Methods Clin Dev 3: 16017.
Merten, O. W. and J. F. Wright (2016). « Towards routine manufacturing of gene therapy drug » Mol Ther Methods Clin Dev 3: 16021.
Richard, I., et al. (2016). « Natural history of LGMD2A for delineating outcome measures in clinical trials. » Ann Clin Transl Neurol 3(4): 248-265.
Richard, I., et al. (2016). « 216th ENMC international workshop: Clinical readiness in FKRP related myopathies » January 15-17, 2016 Naarden, The Netherlands. Neuromuscul Disord 26(10): 717-724.
Rivera-Munoz, P., V. Abramowski, et al. (2016). « Lymphopoiesis in transgenic mice over-expressing Artemis. » Gene Ther 23(2): 176-186.
Verhoeyen, E., et al. (2016). « Twelfth Annual Meeting of the French Society of Cell and Gene Therapy. » Hum Gene Ther 27(7): 555-558.
Ben Abdelwahed Bagga, R., S. Donnou, et al. (2015). « Mouse models of primary central nervous system lymphomas: tools for basing funding and therapeutic strategies. » J Neurooncol 121(1): 9-18.
Cantore, A., M. Ranzani, et al. (2015). « Liver-directed lentiviral gene therapy in a dog model of hemophilia B. » Sci Transl Med 7(277): 277ra228.
Charton, K., J. Sarparanta, et al. (2015). « CAPN3-mediated processing of C-terminal titin replaced by pathological cleavage in titinopathy. » Hum Mol Genet 24(13): 3718-3731.
Cheever, T. R., D. Berkley, et al. (2015). « Perspectives on best practices for gene therapy programs. » Hum Gene Ther 26(3): 127-133.
Cosette, J., A. Moussy, et al. (2015). « Single Cell Dynamics Causes Pareto-Like Effect in Stimulated T Cell Populations. » Sci Rep 5: 17756.
De Cid, R., R. Ben Yaou, et al. (2015). « A new titinopathy: Childhood-juvenile onset Emery-Dreifuss-like phenotype without cardiomyopathy. » Neurology 85(24): 2126-2135.
Droz-Georget Lathion, S., A. Rochat, et al. (2015). « A single epidermal stem cell strategy for safe ex vivo gene therapy. » EMBO Mol Med.
Hacein-Bey Abina, S., H. B. Gaspar, et al. (2015). « Outcomes following gene therapy in patients with severe Wiskott-Aldrich syndrome. » JAMA 313(15): 1550-1563.
Han, S. O., S. Li, et al. (2015). « Enhanced efficacy from gene therapy in Pompe disease using coreceptor blockade. » Hum Gene Ther 26(1): 26-35.
Junge, N., F. Mingozzi, et al. (2015). « Adeno-associated virus vector-based gene therapy for monogenetic metabolic diseases of the liver. » Pediatr Gastroenterol Nutr 60(4): 433-440.
Marini, B., A. Kertesz-Farkas, et al. (2015). « Nuclear architecture dictates HIV-1 integration site selection. » Nature.
Merten, O. W. (2015). « Advances in cell culture: anchorage dependence. » Philos Trans R Soc Lond B Biol Sci 370(1661): 20140040.
Mingozzi, F. and H. Buning (2015). « Adeno-Associated Viral Vectors at the Frontier between Tolerance and Immunity. » Front Immunol 6: 120.

2014

Allenbach, Y., W. Chaara, et al. (2014). « Th1 response and systemic treg deficiency in inclusion body myositis. » PLoS One 9(3): e88788.
Basner-Tschakarjan, E. and F. Mingozzi (2014). « Cell-Mediated Immunity to AAV Vectors, Evolving Concepts and Potential Solutions. » Front Immunol 5: 350.
Brayet, J., F. Zehraoui, et al. (2014). « Towards a piRNA prediction using multiple kernel fusion and support vector machine. » Bioinformatics 30(17): i364-i370.
Childers, M. K., R. Joubert, et al. (2014). « Gene therapy prolongs survival and restores function in murine and canine models of myotubular myopathy. » Sci Transl Med 6(220): 220ra210.
Clermont, D., S. Santoni, et al. (2014). « Assessment of DNA encapsulation, a new room-temperature DNA storage method. » Biopreserv Biobank 12(3): 176-183.
Corre, G., D. Stockholm, et al. (2014). « Stochastic fluctuations and distributed control of gene expression impact cellular memory. » PLoS One 9(12): e115574.
Evila, A., A. Vihola, et al. (2014). « Atypical phenotypes in titinopathies explained by second titin mutations. » Ann Neurol 75(2): 230-240.
Goddard, M. A., E. Burlingame, et al. (2014). « Gait characteristics in a canine model of X-linked myotubular myopathy. » J Neurol Sci 346: 221-226.
Holic, N., A. K. Seye, et al. (2014). « Influence of Mildly Acidic pH Conditions on the Production of Lentiviral and Retroviral Vectors. » Hum Gene Ther Clin Dev 25(3): 178-185.
Moiani, A., J. D. Suerth, et al. (2014). « Genome-wide analysis of alpharetroviral integration in human hematopoietic stem/progenitor cells. » Genes (Basel) 5(2): 415-429.
Mouisel, E., K. Relizani, et al. (2014). « Myostatin is a key mediator between energy metabolism and endurance capacity of skeletal muscle. » Am J Physiol Regul Integr Comp Physiol 307(4): R444-454.
Nathwani, A. C., U. M. Reiss, et al. (2014). « Long-term safety and efficacy of factor IX gene therapy in hemophilia B. » Engl J Med 371(21): 1994-2004.
Neildez-Nguyen, T. M., J. Bigot, et al. (2014). « Hypoxic culture conditions enhance the generation of regulatory T cells. » Immunology.
Rodriguez, E. G., R. Lefebvre, et al. (2014). « Phosphoinositide substrates of myotubularin affect voltage-activated Ca(2)(+) release in skeletal muscle. » Pflugers Arch 466(5): 973-985.
Turchiano, G., M. C. Latella, et al. (2014). « Genomic analysis of sleeping beauty transposon integration in human somatic cells. » PLoS One 9(11): e112712.

2013

Aiuti, A., L. Biasco, et al. (2013). « Lentiviral hematopoietic stem cell gene therapy in patients with Wiskott-Aldrich syndrome. » Science 341(6148): 1233151.
Ben Abdelwahed, R., J. Cosette, et al. (2013). « Lymphoma B-cell responsiveness to CpG-DNA depends on the tumor microenvironment. » J Exp Clin Cancer Res 32: 18.
Biondi, O., M. Villemeur, et al. (2013). « Dual effects of exercise in dysferlinopathy. » Am J Pathol 182(6): 2298-2309.
Cavazza, A., A. Moiani, et al. (2013). « Mechanisms of retroviral integration and mutagenesis. » Hum Gene Ther 24(2): 119-131.
Fayssoil, A., G. Renault, et al. (2013). « Cardiac characterization of mdx mice using high-resolution doppler echocardiography. » J Ultrasound Med 32(5): 757-761.
Fayssoil, A., G. Renault, et al. (2013). « Cardiac Characterization of sgca-Null Mice Using High Resolution Echocardiography. » Neurol Int 5(4): e22.
Kaufmann, K. B., H. Buning, et al. (2013). « Gene therapy on the move. » EMBO Mol Med.
Masat, E., G. Pavani, et al. (2013). « Humoral immunity to AAV vectors in gene therapy: challenges and potential solutions. » Discov Med 15(85): 379-389.
Mingozzi, F., X. M. Anguela, et al. (2013). « Overcoming Preexisting Humoral Immunity to AAV Using Capsid Decoys. » Sci Transl Med 5(194): 194ra192.
Monjaret, F., L. Suel-Petat, et al. (2013). « The phenotype of dysferlin-deficient mice is not rescued by adeno-associated virus-mediated transfer of anoctamin 5. » Hum Gene Ther Clin Dev 24(2): 65-76.
Vignier, N., F. Amor, et al. (2013). « Distinctive serum miRNA profile in mouse models of striated muscular pathologies. » PLoS One 8(2): e55281.

2012

Carrondo, M. J., P. M. Alves, et al. (2012). « How can measurement, monitoring, modeling and control advance cell culture in industrial biotechnology? » Biotechnol J 7(12): 1522-1529.
Donnou, S., C. Galand, et al. (2012). « Murine models of B-cell lymphomas: promising tools for designing cancer therapies. » Adv Hematol 2012: 701704.
Dowling, J. J., R. Joubert, et al. (2012). « Myotubular myopathy and the neuromuscular junction: a novel therapeutic approach from mouse models. » Dis Model Mech 5(6): 852-859.
Guiraud, S., L. van Wittenberghe, et al. (2012). « Identification of decorin derived peptides with a zinc dependent anti-myostatin activity. » Neuromuscul Disord 22(12): 1057-1068.
Marek, M., O. W. Merten, et al. (2012). « Essential C-terminal region of the baculovirus minor capsid protein VP80 binds DNA. » Virol 86(3): 1728-1738.
Mavilio, F. (2012). « Gene therapies need new development models. » Nature 490(7418): 7.
Paldi, A. (2012). « What makes the cell differentiate? » Prog Biophys Mol Biol 110(1): 41-43.
Pathare, A., M. Al Khabori, et al. (2012). « Warfarin pharmacogenetics: development of a dosing algorithm for Omani patients. » Hum Genet 57(10): 665-669.
Sudres, M., S. Cire, et al. (2012). « MyD88 signaling in B cells regulates the production of Th1-dependent antibodies to AAV. » Mol Ther 20(8): 1571-1581.
Yamagata, Y., V. Parietti, et al. (2012). « Lentiviral transduction of CD34(+) cells induces genome-wide epigenetic modifications. » PLoS One 7(11): e48943.
Yamagata, Y., P. Szabo, et al. (2012). « Rapid turnover of DNA methylation in human cells. » Epigenetics 7(2): 141-145.

2011

Barde, I., E. Laurenti, et al. (2011). « Lineage- and stage-restricted lentiviral vectors for the gene therapy of chronic granulomatous disease. » Gene Ther 18(11): 1087-1097.
Bechinger, B., V. Vidovic, et al. (2011). « A new family of peptide-nucleic acid nanostructures with potent transfection activities. » J Pept Sci 17(2): 88-93.
Brun-Graeppi, A. K., C. Richard, et al. (2011). « Cell microcarriers and microcapsules of stimuli-responsive polymers. » J Control Release 149(3): 209-224.
Carre-Pierrat, M., A. Lafoux, et al. (2011). « Pre-clinical study of 21 approved drugs in the mdx mouse. » Neuromuscul Disord 21(5): 313-327.
Carrondo, M., A. Panet, et al. (2011). « Integrated strategy for the production of therapeutic retroviral vectors. » Hum Gene Ther 22(3): 370-379.
Galy, A. and A. J. Thrasher (2011). « Gene therapy for the Wiskott-Aldrich syndrome. » Curr Opin Allergy Clin Immunol 11(6): 545-550.
Guiraud, S., D. Alimi-Guez, et al. (2011). « The reverse block copolymer Pluronic 25R2 promotes DNA transfection of skeletal muscle. » Macromol Biosci 11(5): 590-594.
Jeanson-Leh, L., S. Charrier, et al. (2011). « The c.273+11dup genetic change in the WAS gene is a functionally neutral polymorphism. » Eur J Haematol 87(4): 366-371.
Kamen, A. A., M. G. Aucoin, et al. (2011). « An initiative to manufacture and characterize baculovirus reference material. » J Invertebr Pathol 107 Suppl: S113-117.
Mezzina, M. and O. W. Merten (2011). « Adeno-associated viruses. » Methods Mol Biol 737: 211-234.
Sambasivan, R., R. Yao, et al. (2011). « Pax7-expressing satellite cells are indispensable for adult skeletal muscle regeneration. » Development 138(17): 3647-3656.
Stacey, G. N. and O. W. Merten (2011). « Host cells and cell banking. » Methods Mol Biol 737: 45-88.

2010

Beggs, A.H., J. Bohm, E. Snead, M. Kozlowski, M. Maurer, K. Minor, M.K. Childers, S.M. Taylor, C. Hitte, J.R. Mickelson, L.T. Guo, A.P. Mizisin, A. Buj-Bello, L. Tiret, J. Laporte, and G.D. Shelton, « MTM1 mutation associated with X-linked myotubular myopathy in Labrador Retrievers » Proc Natl Acad Sci U S A, 2010. 107(33): p. 14697-14702
Benkhelifa-Ziyyat, S., S. Bucher, M.A. Zanta-Boussif, J. Pasquet, and O. Danos, « Changes in the accessibility of the HIV-1 Integrase C-terminus in the presence of cellular proteins » Retrovirology, 2010. 7(1): p. 27
Bolduc, V., G. Marlow, K.M. Boycott, K. Saleki, H. Inoue, J. Kroon, M. Itakura, Y. Robitaille, L. Parent, F. Baas, K. Mizuta, N. Kamata, I. Richard, W.H. Linssen, I. Mahjneh, M. de Visser, R. Bashir, and B. Brais, « Recessive Mutations in the Putative Calcium-Activated Chloride Channel Anoctamin 5 Cause Proximal LGMD2L and Distal MMD3 Muscular Dystrophies » Am J Hum Genet, 2010. 86(2): p. 213-221
Boutin, S., V. Monteilhet, P. Veron, C. Leborgne, O. Benveniste, M.F. Montus, and C. Masurier, « Prevalence of serum IgG and neutralizing factors against adeno-associated virus types 1, 2, 5, 6, 8 and 9 in the healthy population: implications for gene therapy using AAV vectors » Hum Gene Ther, 2010. 21(6): p. 704-712
Charton, K., N. Daniele, A. Vihola, C. Roudaut, E. Gicquel, F. Monjaret, A. Tarrade, J. Sarparanta, B. Udd, and I. Richard, « Removal of the calpain 3 protease reverses the myopathology in a mouse model for titinopathies » Hum Mol Genet, 2010. 19(23): p. 4608-4624
Capron, C., C. Lacout, Y. Lecluse, V. Jalbert, H. Chagraoui, S. Charrier, A. Galy, A. Bennaceur-Griscelli, E. Cramer-Borde, and W. Vainchenker, « A major role of TGF-{beta}1 in the homing capacities of murine hematopoietic stem cell/ progenitors » Blood, 2010. 116(8): p. 1244-1253
Claeys, K.G., M. Sozanska, J.J. Martin, E. Lacene, L. Vignaud, D. Stockholm, P. Laforet, B. Eymard, A. Kichler, D. Scherman, T. Voit, and D. Israeli, « DNAJB2 Expression in Normal and Diseased Human and Mouse Skeletal Muscle » Am J Pathol, 2010. 176(6): p. 2901-2910
Hauser, H., M. Carrondo, D. Wirth, A.S. Coroadinha, P. Cruz, H. Falk, R. Schucht, F. Dupont, C. Geny-Fiamma, and O.W. Merten, « Integrated strategy for the production of therapeutic retroviral vectors » Hum Gene Ther, 2010. <em>Online Ahead of Editing: November 2</em>
Klein, E., M. Ciobanu, J. Klein, V. Machi, C. Leborgne, T. Vandamme, B. Frisch, F. Pons, A. Kichler, G. Zuber, and L. Lebeau, « HFP Fluorinated Cationic Lipids for Enhanced Lipoplex Stability and Gene Delivery » Bioconjug Chem, 2010. 21(2): p. 360-71
Klein, E., C. Leborgne, M. Ciobanu, J. Klein, B. Frisch, F. Pons, G. Zuber, D. Scherman, A. Kichler, and L. Lebeau, « Nucleic acid transfer with hemifluorinated polycationic lipids » Biomaterials, 2010. 31(17): p. 4781-8
Krahn, M., N. Wein, M. Bartoli, W. Lostal, S. Courrier, N. Bourg-Alibert, K. Nguyen, C. Vial, N. Streichenberger, V. Labelle, D. DePetris, C. Pécheux, F. Leturcq, P. Cau, I. Richard, and N. Lévy, « A Naturally Occurring Human Minidysferlin Protein Repairs Sarcolemmal Lesions in a Mouse Model of Dysferlinopathy » Science Translational Medicine, 2010. 2(50): p. 50ra69
Lan, Y., B. Langlet-Bertin, V. Abbate, L.S. Vermeer, X. Kong, K.E. Sullivan, C. Leborgne, D. Scherman, R.C. Hider, A.F. Drake, S.S. Bansal, A. Kichler, and A.J. Mason, « Incorporation of 2,3-Diaminopropionic Acid into Linear Cationic Amphipathic Peptides Produces pH-Sensitive Vectors » Chembiochem, 2010. 11(9): p. 1266 – 1272
Langlet-Bertin, B., C. Leborgne, D. Scherman, B. Bechinger, A.J. Mason, and A. Kichler, « Design and Evaluation of Histidine-Rich Amphipathic Peptides for siRNA Delivery » Pharm Res, 2010. 27(7): p. 1426-1436
Laure, L., N. Daniele, L. Suel, S. Marchand, S. Aubert, N. Bourg, C. Roudaut, S. Duguez, M. Bartoli, and I. Richard, « A new pathway encompassing calpain 3 and its newly identified substrate cardiac ankyrin repeat protein is involved in the regulation of the nuclear factor-kappaB pathway in skeletal muscle » FEBS J, 2010. 277(20): p. 4322-4337
Lidgi-Guigui, N., C. Guis, B. Brissault, A. Kichler, C. Leborgne, D. Scherman, S. Labdi, and P.A. Curmi, « Investigation of DNA Condensing Properties of Amphiphilic Triblock Cationic Polymers by Atomic Force Microscopy » Langmuir, 2010. 26(22): p. 17552-17557
Lock, M., S. McGorray, A. Auricchio, E. Ayuso, E.J. Beecham, V. Blouin-Tavel, F. Bosch, M. Bose, B.J. Byrne, T. Caton, J.A. Chiorini, A. Chtarto, K.R. Clark, T. Conlon, C. Darmon, M. Doria, A. Douar, T.R. Flotte, J.D. Francis, A. Francois, M. Giacca, M.T. Korn, I. Korytov, X. Leon, B. Leuchs, G. Lux, C. Melas, H. Mizukami, P. Moullier, M. Müller, K. Ozawa, T. Philipsberg, K. Poulard, C. Raupp, C. Rivière, S.D. Roosendaal, R.J. Samulski, S.M. Soltys, R. Surosky, L. Tenenbaum, D.L. Thomas, B. van Montfort, G. Veres, J.F. Wright, Y. Xu, O. Zelenaia, L. Zentilin, and R.O. Snyder, « Characterization of a Recombinant Adeno-Associated Virus Type 2 Reference Standard Material » Human Gene Therapy, 2010. 21(10): p. 1273-1285
Lostal, W., M. Bartoli, N. Bourg, C. Roudaut, A. Bentaib, K. Miyake, N. Guerchet, F. Fougerousse, P. McNeil, and I. Richard, « Efficient recovery of dysferlin deficiency by dual adeno-associated vectors mediated gene transfer » Hum. Mol. Genet., 2010. 19(10): p. 1897-1907
Moulay, G., C. Masurier, P. Bigey, D. Scherman, and A. Kichler, « Soluble TNF-[alpha] receptor secretion from healthy or dystrophic mice after AAV6-mediated muscle gene transfer » Gene Ther, 2010. 17(11): p. 1400–1410
Sarparanta, J., G. Blandin, K. Charton, A. Vihola, S. Marchand, A. Milic, P. Hackman, E. Ehler, I. Richard, and B. Udd, « Interactions with M-band titin and calpain 3 link myospryn (CMYA5) to tibial and limb-girdle muscular dystrophies » J Biol Chem, 2010. 285(39): p. 30304-30315
Schweizer, M. and O.W. Merten, « Large-Scale Production Means for the Manufacturing of Lentiviral Vectors » Curr Gene Ther, 2010. 10(6): p. 474-486
Wasungu, L., A.L. Marty, M.F. Bureau, A. Kichler, M. Bessodes, J. Teissie, D. Scherman, M.P. Rols, and N. Mignet, « Pre-treatment of cells with pluronic L64 increases DNA transfection mediated by electrotransfer » J Control Release, 2010. 149(2): p. 117-125.]]></content:encoded>

2009

Andriola Silva, A.K., Richard, C., Bessodes, M., Scherman, D. and Merten, O.-W.: « Growth Factor Delivery Approaches in Hydrogels » Biomacromolecules 10 (2009) 9-18.
Bouazza, B., Kratassiouk, G., Gjata, B., Perie, S., Guily, J.L., Butler-Browne, G.S. andSvinartchouk, F.: « Analysis of growth factor expression in affected and unaffected muscles of oculo-pharyngeal muscular dystrophy (OPMD) patients: A pilot study » Neuromuscul Disord 19 (2009) 199-206.
Ciron, C., Cressant, A., Roux, F., Raoul, S., Cherel, Y., Hantraye, P., Deglon, N., Schwartz, B.,Barkats, M., Heard, J.M., Tardieu, M., Moullier, P. and Colle, M.A.: « Human alpha-Iduronidase Gene Transfer Mediated by Adeno-Associated Virus Types 1, 2, and 5 in the Brain of Nonhuman Primates: Vector Diffusion and Biodistribution » Hum Gene Ther 20 (2009) 350-360.
Denard, J., Rundwasser, S., Laroudie, N., Gonnet, F., Naldini, L., Radrizzani, M., Galy, A., Merten, O.-W., Danos, O. and Svinartchouk, F.: « Quantitative proteomic analysis of lentiviral vectors using two dimensional gel electrophoresis » Proteomics 9 (2009) 3666-3676.
Duque, S., Joussemet, B., Riviere, C., Marais, T., Dubreil, L., Douar, A.M., Fyfe, J., Moullier, P., Colle, M.A. and Barkats, M.: « Intravenous Administration of Self-complementary AAV9 Enables Transgene Delivery to Adult Motor Neurons » Mol Ther 17 (2009) 1187–1196.
Gérard, A., Ghiotto, M., Fos, C., Guittard, G., Compagno, D., Galy, A., Lemay, S., Olive, D. and Nunès, J.A.: « Dok-4 is a novel negative regulator of T cell activation » J Immunol 182 (2009) 7681-7689.
Gerard, X., Vignaud, L., Charkes, S., Pinset, C., Scherman, D., Kichler, A. and Israeli, D.: « Real-time monitoring of cell transplantation in mouse dystrophic muscles by a secreted alkaline phosphatase reporter gene » Gene Ther 16 (2009) 815–819.
Goizet, C., Boukhris, A., Durr, A., Beetz, C., Truchetto, J., Tesson, C., Tsaousidou, M., Forlani, S., Guyant-Marechal, L., Fontaine, B., Guimaraes, J., Isidor, B., Chazouilleres, O., Wendum, D.,Grid, D., Chevy, F., Chinnery, P.F., Coutinho, P., Azulay, J.P., Feki, I., Mochel, F., Wolf, C., Mhiri, C., Crosby, A., Brice, A. and Stevanin, G.: « CYP7B1 mutations in pure and complex forms of hereditary spastic paraplegia type 5 » Brain 132 (2009) 1589-600.
Jedidi, A., Marty, C., Oligo, C., Jeanson-Leh, L., Ribeil, J.A., Casadevall, N., Galy, A., Vainchenker, W. and Villeval, J.L.: « Selective reduction of JAK2V617F-dependent cell growth by siRNA / shRNA and its reversal by cytokines » Blood 114 (2009) 1842-1851.
Laure, L., Suel, L., Roudaut, C., Bourg, N., Ouali, A., Bartoli, M., Richard, I. and Daniele, N.: « Cardiac ankyrin repeat protein is a marker of skeletal muscle pathological remodelling » Febs J 276 (2009) 669-84.
Le Roy, F., Charton, K., Lorson, C.L. and Richard, I.: « RNA-targeting approaches for neuromuscular diseases » Trends Mol Med Available online 10 November (2009).
Mantovani, J., Charrier, S., Eckenberg, R., Saurin, W., Danos, O., Perea, J. and Galy, A.: « Diverse genomic integration of a lentiviral vector developed for the treatment of Wiskott-Aldrich syndrome » J Gene Med 11 (2009) 645 – 654.
Marangoni, F., Bosticardo, M., Charrier, S., Draghici, E., Locci, M., Scaramuzza, S., Panaroni, C., Ponzoni, M., Sanvito, F., Doglioni, C., Liabeuf, M., Gjata, B., Montus, M., Siminovitch, K., Aiuti, A., Naldini, L., Dupre, L., Roncarolo, M.G., Galy, A. and Villa, A.: « Evidence for Long-term Efficacy and Safety of Gene Therapy for Wiskott-Aldrich Syndrome in Preclinical Models » Mol Ther 17 (2009) 1073–1082.
Megarbane, H., Florence, J., Oliver Sass, J., Schwonbeck, S., Foglio, M., de Cid, R., Cure, S.,Saker, S., Megarbane, A. and Fischer, J.: « An Autosomal-Recessive Form of Cutis Laxa Is Due to Homozygous Elastin Mutations, and the Phenotype May Be Modified by a Heterozygous Fibulin 5 Polymorphism » J Invest Dermatol 129 (2009) 1650–1655.
Mellgren, R.L., Miyake, K., Kramerova, I., Spencer, M.J., Bourg, N., Bartoli, M., Richard, I., Greer, P.A. and McNeil, P.L.: « Calcium-dependent plasma membrane repair requires m- or mu-calpain, but not calpain-3, the proteasome, or caspases » Biochim Biophys Acta Available online 23 September (2009).
Modlich, U., Navarro, S., Zychlinski, D., Maetzig, T., Knoess, S., Brugman, M.H., Schambach, A.,Charrier, S., Galy, A., Thrasher, A., Bueren, J. and Baum, C.: « Insertional transformation of hematopoietic cells by selfinactivating lentiviral and gammaretroviral vectors » Mol Ther (Published online 11 August 2009).
Oudot, T., Lesueur, F., Guedj, M., de Cid, R., McGinn, S., Heath, S., Foglio, M., Prum, B., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « An Association Study of 22 Candidate Genes in Psoriasis Families Reveals Shared Genetic Factors with Other Autoimmune and Skin Disorders » J Invest Dermatol Published online 25 June (2009).
Tazir, M., Ali-Pacha, L., M'Zahem, A., Delaunoy, J.P., Fritsch, M., Nouioua, S., Benhassine, T., Assami, S., Grid, D., Vallat, J.M., Hamri, A. and Koenig, M.: « Ataxia with oculomotor apraxia type 2: A clinical and genetic study of 19 patients » J Neurol Sci 278 (2009) 77-81.
Tazir, M., Nouioua, S., Magy, L., Huehne, K., Assami, S., Urtizberea, A., Grid, D., Hamadouche, T., Rautenstrauss, B. and Vallat, J.-M.: « Phenotypic variability in giant axonal neuropathy » Neuromuscular Disorders 19 (2009) 270-274.
Verburg, E., Murphy, R.M., Richard, I. and Lamb, G.D.: « Involvement of calpains in Ca2+-induced disruption of excitation-contraction coupling in mammalian skeletal muscle fibers » Am J Physiol Cell Physiol 296 (2009) C1115-C1122.
Veron, P., Boutin, S., Martin, S., Chaperot, L., Plumas, J., Davoust, J. and Masurier, C.: « Highly efficient transduction of human plasmacytoid dendritic cells without phenotypic and functional maturation » J Transl Med 7 (2009) 10.
Zanta-Boussif, M.A., Charrier, S., Brice-Ouzet, A., Martin, S., Opolon, P., Thrasher, A.J., Hope, T.J. and Galy, A.: « Validation of a mutated PRE sequence allowing high and sustained transgene expression while abrogating WHV-X protein synthesis: application to the gene therapy of WAS » Gene Ther 16 (2009) 605–619.

2008

Agbulut, O., Vignaud, A., Hourde, C., Mouisel, E., Fougerousse, F., Butler-Browne, G. and Ferry, A.: « Slow myosin heavy chain expression in the absence of muscle activity » Am J Physiol Cell Physiol Published Online October 22 2008.
Amaral, A.I., Coroadinha, A.S., Merten, O.W. and Alves, P.M.: « Improving retroviral vectors production: Role of carbon sources in lipid biosynthesis » J Biotechnol 138 (2008) 57-66.
Bacquet, C., Imamura, T., Gonzalez, C.A., Conejeros, I., Kausel, G., Neildez-Nguyen, T.M.,Paldi, A. and Gallardo, M.H.: « Epigenetic processes in a tetraploid mammal » Mamm Genome 19 (2008) 439-447.
Bartoli, M., Gicquel, E., Barrault, L., Soheili, T., Malissen, M., Malissen, B., Vincent-lacaze, N., Perez, N., Udd, B., Danos, O. and Richard, I.: « Mannosidase I inhibition rescues the human {alpha}-sarcoglycan R77C recurrent mutation » Hum. Mol. Genet. 17 (2008) 1214-1221.
Benayoun, B., Baghdiguian, S., Lajmanovich, A., Bartoli, M., Daniele, N., Gicquel, E., Bourg, N., Raynaud, F., Pasquier, M.A., Suel, L., Lochmuller, H., Lefranc, G. and Richard, I.: « NF-{kappa}B-dependent expression of the antiapoptotic factor c-FLIP is regulated by calpain 3, the protein involved in limb-girdle muscular dystrophy type 2A » Faseb J 22 (2008) 1521-1529.
Benjelloun, F., Garrigue, A., Demerens-de Chappedelaine, C., Soulas-Sprauel, P., Malassis-Seris, M., Stockholm, D., Hauer, J., Blondeau, J., Riviere, J., Lim, A., Le Lorc'h, M., Romana, S., Brousse, N., Paques, F., Galy, A., Charneau, P., Fischer, A., de Villartay, J.P. and Cavazzana-Calvo, M.: « Stable and Functional Lymphoid Reconstitution in Artemis-deficient Mice Following Lentiviral Artemis Gene Transfer Into Hematopoietic Stem Cells » Mol Ther 16 (2008) 490–1499.
Boguslaw, A.K., Kirillova, I., Richard, R.E., Israeli, D. and Yablonka-Reuveni, Z.: « FGFR4 and its novel splice form in myogenic cells: interplay of glycosylation and tyrosine phosphorylation » J Cell Physiol 215 (2008) 803 – 817.
Buj-Bello, A., Fougerousse, F., Schwab, Y., Messaddeq, N., Spehner, D., Pierson, C.R., Durand, M., Kretz, C., Danos, O., Douar, A.M., Beggs, A.H., Schultz, P., Montus, M., Denefle, P. and Mandel, J.L.: « AAV-mediated intramuscular delivery of myotubularin corrects the myotubular myopathy phenotype in targeted murine muscle and suggests a function in plasma membrane homeostasis » Hum Mol Genet 17 (2008) 2132-2143.
Claus, S., Fischer, J., Megarbane, H., Megarbane, A., Jobard, F., Debret, R., Peyrol, S., Saker, S., Devillers, M., Sommer, P. and Damour, O.: « A p.C217R Mutation in Fibulin-5 from Cutis Laxa Patients Is Associated with Incomplete Extracellular Matrix Formation in a Skin Equivalent Model » J Invest Dermatol 128 (2008) 1442–1450.
Coulis, G., Becila, S., Herrera-Mendez, C.H., Sentandreu, M.A., Raynaud, F., Richard, I., Benyamin, Y. and Ouali, A.: « Calpain 1 binding capacities of the N1-line region of titin are significantly enhanced by physiological concentrations of calcium » Biochemistry 47 (2008) 9174-83.
Delaguillaumie, A., Marin-Esteban, V., Setterblad, N., Jeanson-Leh, L., Assier, E., Gelin, C., Charron, D., Galy, A. and Mooney, N.: « Contrasting cytoskeletal regulation of MHC class II peptide presentation by human B cells or dendritic cells » Eur J Immunol 38 (2008) 1096-1105.
Delluc-Clavieres, A., Le Bec, C., Van den Berghe, L., Conte, C., Allo, V., Danos, O. and Prats, A.C.: « Efficient gene transfer in skeletal muscle with AAV-derived bicistronic vector using the FGF-1 IRES » Gene Ther 15 (2008) 1090–1098.
Galy, A., Roncarolo, M.G. and Thrasher, A.J.: « Development of lentiviral gene therapy for Wiskott Aldrich syndrome » Expert Opin Biol Ther 8 (2008) 181-190.
Gonnet, F., Bouazza, B., Millot, G.A., Ziaei, S., Garcia, L., Butler-Browne, G.S., Mouly, V., Tortajada, J., Danos, O. and Svinartchouk, F.: « Proteome analysis of differentiating human myoblasts by dialysis-assisted two-dimensional gel electrophoresis (DAGE) » Proteomics 8 (2008) 264-278.
Hackman, P., Marchand, S., Sarparanta, J., Vihola, A., Penisson-Besnier, I., Eymard, B., Pardal-Fernandez, J.M., Hammouda, E.H., Richard, I., Illa, I. and Udd, B.: « Truncating mutations in C-terminal titin may cause more severe tibial muscular dystrophy (TMD) » Neuromuscul Disord Published online 22 October 2008.
Hamadouche, T., Poitelon, Y., Genin, E., Chaouch, M., Tazir, M., Kassouri, N., Nouioua, S., Chaouch, A., Boccaccio, I., Benhassine, T., De Sandre-Giovannoli, A., Grid, D., Levy, N. and Delague, V.: « Founder Effect and Estimation of the Age of the c.892C&gt;T (p.Arg298Cys) Mutation in LMNA Associated to Charcot-Marie-Tooth Subtype CMT2B1 in Families from North Western Africa » Ann Hum Genet (2008).
Khoury, M., Escriou, V., Courties, G., Galy, A., Yao, R., Largeau, C., Scherman, D., Jorgensen, C. and Apparailly, F.: « Efficient suppression of murine arthritis by combined anticytokine small interfering RNA lipoplexes » Arthritis Rheum 58 (2008) 2356-2367.
Krahn, M., Beroud, C., Labelle, V., Nguyen, K., Bernard, R., Bassez, G., Figarella-Branger, D., Fernandez, C., Bouvenot, J., Richard, I., Ollagnon-Roman, E., Bevilacqua, J.A., Salvo, E., Attarian, S., Chapon, F., Pellissier, J.F., Pouget, J., Hammouda, E.H., Laforet, P., Urtizberea, J.A., Eymard, B., Leturcq, F. and Levy, N.: « Analysis of the DYSF mutational spectrum in a large cohort of patients » Hum Mutat Published Online 13 October 2008.
Lorain, S., Gross, D.A., Goyenvalle, A., Danos, O., Davoust, J. and Garcia, L.: « Transient Immunomodulation Allows Repeated Injections of AAV1 and Correction of Muscular Dystrophy in Multiple Muscles » Mol Ther 16 (2008) 541–547.
Mason, A.J., Moussaoui, W., Abdelrahman, T., Boukhari, A., Bertani, P., Marquette, A., Shooshtarizaheh, P., Moulay, G., Boehm, N., Guerold, B., Sawers, R.J., Kichler, A., Metz-Boutigue, M.H., Candofi, E., Prevost, G. and Bechinger, B.: « Structural determinants of antimicrobial and antiplasmodial activity and selectivity in histidine rich amphipathic cationic peptides » J Biol Chem Published online November 4, 2008.
Neildez-Nguyen, T.M., Parisot, A., Vignal, C., Rameau, P., Stockholm, D., Picot, J., Allo, V., Le Bec, C., Laplace, C. and Paldi, A.: « Epigenetic gene expression noise and phenotypic diversification of clonal cell populations » Differentiation 76 (2008) 33-40.
Pomel, C., Leborgne, C., Cheradame, H., Scherman, D., Kichler, A. and Guegan, P.: « Synthesis and Evaluation of Amphiphilic Poly(tetrahydrofuran-b-ethylene oxide) Copolymers for DNA Delivery into Skeletal Muscle » Pharm Res Published online 4 September 2008.
Vignaud, A., Fougerousse, F., Mouisel, E., Bertrand, C., Bonafos, B., Molgo, J., Ferry, A. and Chatonnet, A.: « Genetic ablation of acetylcholinesterase alters muscle function in mice » Chem Biol Interact 175 (2008) 129-130.
Vignaud, A., Fougerousse, F., Mouisel, E., Guerchet, N., Hourde, C., Bacou, F., Butler-Browne, G.S., Chatonnet, A. and Ferry, A.: « Genetic inactivation of acetylcholinesterase causes functional and structural impairment of mouse soleus muscles » Cell Tissue Res 333 (2008) 289-296.

2007

Attali, M., Stetsyuk, V., Basmaciogullari, A., Aiello, V., Zanta-Boussif, M.A., Duvillie, B. and Scharfmann, R.: « Control of beta-cell differentiation by the pancreatic mesenchyme » Diabetes 56 (2007) 1248-58.
Bartoli, M., Poupiot, J., Vulin, A., Fougerousse, F., Arandel, L., Daniele, N., Roudaut, C., Noulet, F., Garcia, L., Danos, O. and Richard, I.: « AAV-mediated delivery of a mutated myostatin propeptide ameliorates calpain 3 but not alpha-sarcoglycan deficiency » Gene Ther 14 (2007) 733–740.
Benchaouir, R., Meregalli, M., Farini, A., D'Antona, G., Belicchi, M., Goyenvalle, A., Battistelli, M., Bresolin, N., Bottinelli, R., Garcia, L. and Torrente, Y.: « Restoration of Human Dystrophin Following Transplantation of Exon-Skipping-Engineered DMD Patient Stem Cells into Dystrophic Mice » Cell Stem Cell 1 (2007) 646.
Benchaouir, R., Picot, J., Greppo, N., Rameau, P., Stockholm, D., Garcia, L., Paldi, A. and Laplace-Builhe, C.: « Combination of quantification and observation methods for study of ”side population” cells in their “in vitro” microenvironment. » Cytometry A 71 (2007) 251-7.
Beroud, C., Tuffery-Giraud, S., Matsuo, M., Hamroun, D., Humbertclaude, W., Monnier, N., Moizard, M.P., Voelckel, M.A., Calemard, L.M., Boisseau, P., Blayau, M., Philippe, C., Cossee, M., Pages, M., Rivier, F., Danos, O., Garcia, L. and Claustres, M.: « Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63 % of patients with Duchenne muscular dystrophy » Hum Mutat 28 (2007) 196-202.
Brissault, B., Kichler, A., Leborgne, C., Jarroux, N., Cheradame, H. and Guis, C.: « Amphiphilic Poly[(propylene glycol)-block-(2-methyl-2-oxazoline)] Copolymers for Gene Transfer in Skeletal Muscle » ChemMedChem 2 (2007) 1202-1207.
Carmignac, V., Salih, M.A., Quijano-Roy, S., Marchand, S., Al Rayess, M.M., Mukhtar, M.M., Urtizberea, J.A., Labeit, S., Guicheney, P., Leturcq, F., Gautel, M., Fardeau, M., Campbell, K.P.,Richard, I., Estournet, B. and Ferreiro, A.: « C-terminal titin deletions cause a novel early-onset myopathy with fatal cardiomyopathy » Ann Neurol 61 (2007) 340-351.
Charrier, S., Dupre, L., Scaramuzza, S., Jeanson-Leh, L., Blundell, M.P., Danos, O., Cattaneo, F., Aiuti, A., Eckenberg, R., Thrasher, A.J., Roncarolo, M.G. and Galy, A.: « Lentiviral vectors targeting WASp expression to hematopoietic cells, efficiently transduce and correct cells from WAS patients » Gene Ther 14 (2007) 415-28.
Coecke, S., Balls, M., Bowe, G., Davis, J., Gstraunthaler, G., Hartung, T., Hay, R., Price, A.,Merten, O.-W., Stokes, W., Schlechtman, L. and Stacey, G.: « Guidance on Good Cell Culture Practice. A report of the second ECVAM task force on Good Cell Culture Practice – Proceedings of the 19th ESACT Meeting, Harrogate, UK, June 5-8, 2005 » In: Smith, R. (Ed.), Cell Technology for Cell Products. Springer, Dordrecht, 2007, pp. 313-315.
Daniele, N., Richard, I. and Bartoli, M.: « Ins and outs of therapy in limb girdle muscular dystrophies » Int J Biochem Cell Biol 39 (2007) 1608-24.
Deburgrave, N., Daoud, F., Llense, S., Barbot, J.C., Recan, D., Peccate, C., Burghes, A.H.M., Beroud, C., Garcia, L., Kaplan, J., Chelly, J. and Leturcq, F.: « Protein, and mRNABased phenotype-genotype correlations in DMD/DMD with point mutations and molecular basis for BMD with nonsense and frameshift mutations in the DMD gene » Hum Mutat 28 (2007) 183-195.
Delague, V., Jacquier, A., Hamadouche, T., Poitelon, Y., Baudot, C., Boccaccio, I., Chouery, E., Chaouch, M., Kassouri, N., Jabbour, R., Grid, D., Megarbane, A., Haase, G. and Levy, N.: « Mutations in FGD4 encoding the Rho GDP/GTP exchange factor FRABIN cause autosomal recessive Charcot-Marie-Tooth type 4H » Am J Hum Genet 81 (2007) 1-16.
Delenda, C., Chillon, M., Douar, A.M. and Merten, O.-W.: « Cells for gene therapy and vector production » In: Pörtner, R. (Ed.), Animal Cell Biotechnology: Methods and Protocols. Humana Press, Totowa, 2007, pp. 23-91.
Elleuch, N., Bouslam, N., Hanein, S., Lossos, A., Hamri, A., Klebe, S., Meiner, V., Birouk, N., Lerer, I., Grid, D., Bacq, D., Tazir, M., Zelenika, D., Argov, Z., Durr, A., Yahyaoui, M., Benomar, A., Brice, A. and Stevanin, G.: « Refinement of the SPG15 candidate interval and phenotypic heterogeneity in three large Arab families » Neurogenetics (2007).
Faure, O., Graff-Dubois, S., Alves, P.M., Cornet, S., Duffour, M.T., Scardino, A., Gross, D.A., Miconnet, I., Salcedo, M., Chouaib, S., Lemonnier, F.A., Abastado, J.P. and Kosmatopoulos, K.: « Induction of multiple CD8+ T cell responses against the inducible Hsp70 employing an Hsp70 oligoepitope peptide » Oncol Rep 17 (2007) 679-85.
Fougerousse, F., Bartoli, M., Poupiot, J., Arandel, L., Durand, M., Guerchet, N., Gicquel, E., Danos, O. and Richard, I.: « Phenotypic Correction of alpha-Sarcoglycan Deficiency by Intra-arterial Injection of a Muscle-specific Serotype 1 rAAV Vector » Mol Ther 15 (2007) 53-61.
Griggs, R., Vihola, A., Hackman, P., Talvinen, K., Haravuori, H., Faulkner, G., Eymard, B.,Richard, I., Selcen, D., Engel, A., Carpen, O. and Udd, B.: « Zaspopathy in a large classic late-onset distal myopathy family » Brain 130 (2007) 1477-84.
Israeli, D., Ziaei, S., Gjata, B., Benchaouir, R., Rameau, P., Marais, T., Fukada, S.I., Segawa, M., Yamamoto, H., Gonin, P., Danos, O. and Garcia, L.: « Expression of mdr1 is required for efficient long term regeneration of dystrophic muscle » Exp Cell Res 313 (2007) 2438-50.
Jaiswal, J.K., Marlow, G., Summerill, G., Mahjneh, I., Mueller, S., Hill, M., Miyake, K., Haase, H., Anderson, L.V., Richard, I., Kiuru-Enari, S., McNeil, P.L., Simon, S.M. and Bashir, R.: « Patients with a non-dysferlin Miyoshi myopathy have a novel membrane repair defect » Traffic 8 (2007) 77-88.
Jeanson-Leh, L., Blondeau, J. and Galy, A.: « Optimization of short hairpin RNA for lentiviral-mediated RNAi against WAS » Biochem Biophys Res Commun 362 (2007) Pages 498-503.
Kichler, A., Leborgne, C., Danos, O. and Bechinger, B.: « Characterization of the gene transfer process mediated by histidine-rich peptides » J Mol Med 85 (2007) 191-201.
Klebe, S., Durr, A., Bouslam, N., Grid, D., Paternotte, C., Depienne, C., Hanein, S., Bouhouche, A., Elleuch, N., Azzedine, H., Poea-Guyon, S., Forlani, S., Denis, E., Charon, C., Hazan, J., Brice, A. and Stevanin, G.: « Spastic paraplegia 5: Locus refinement, candidate gene analysis and clinical description » Am J Med Genet B Neuropsychiatr Genet (2007).
Lesueur, F., Bouadjar, B., Lefevre, C., Jobard, F., Audebert, S., Lakhdar, H., Martin, L., Tadini, G., Karaduman, A., Emre, S., Saker, S., Lathrop, M. and Fischer, J.: « Novel mutations in ALOX12B in patients with autosomal recessive congenital ichthyosis and evidence for genetic heterogeneity on chromosome 17p13 » J Invest Dermatol 127 (2007) 829-34.
Lesueur, F., Lefevre, C., Has, C., Guilloud-Bataille, M., Oudot, T., Mahe, E., Lahfa, M., Mansouri, S., Mosharraf-Olmolk, H., Sobel, E., Heath, S., Lathrop, M., Dizier, M.H., Prud'Homme, J.F. and Fischer, J.: « Confirmation of psoriasis susceptibility loci on chromosome 6p21 and 20p13 in French families » J Invest Dermatol 127 (2007) 1403-9.
Lesueur, F., Oudot, T., Heath, S., Foglio, M., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « ADAM33, a New Candidate for Psoriasis Susceptibility » PLoS ONE 2 (2007) e906.
Mars, L.T., Bauer, J., Gross, D.A., Bucciarelli, F., Firat, H., Hudrisier, D., Lemonnier, F., Kosmatopoulos, K. and Liblau, R.S.: « CD8 T Cell Responses to Myelin Oligodendrocyte Glycoprotein-Derived Peptides in Humanized HLA-A*0201-Transgenic Mice » J Immunol 179 (2007) 5090-8.
Mason, A.J., Bechinger, B. and Kichler, A.: « Rational design of vector and antibiotic peptides using solid-state NMR. » Mini Rev Med Chem 7 (2007) 491-7.
Mason, A.J., Bertani, P., Moulay, G., Marquette, A., Perrone, B., Drake, A.F., Kichler, A. and Bechinger, B.: « Membrane Interaction of Chrysophsin-1, a Histidine-Rich Antimicrobial Peptide from Red Sea Bream » Biochemistry (2007).
Mason, A.J., Leborgne, C., Moulay, G., Martinez, A., Danos, O., Bechinger, B. and Kichler, A.: « Optimising histidine rich peptides for efficient DNA delivery in the presence of serum » J Control Release 118 (2007) 95-104.
Masurier, C., Boutin, S., Veron, P., Bernard, J., Danos, O. and Davoust, J.: « Enhanced lentiviral transduction of monocyte-derived dendritic cells in presence of conditioned medium from dying monocytes » Hum Gene Ther? (2007) 161 -170.
Maystadt, I., Rezsöhazy, R., Barkats, M., Duque, S., Vannuffel, P., Remacle, S., Lambert, B., Najimi, M., Sokal, E., Munnich, A., Viollet, L. and Verellen-Dumoulin, C.: « The Nuclear Factor B–Activator Gene PLEKHG5 Is Mutated in a Form of Autosomal Recessive Lower Motor Neuron Disease with Childhood Onset » Am J Hum Genet 81 (2007) 67-76.
Milic, A., Daniele, N., Lochmuller, H., Mora, M., Comi, G.P., Moggio, M., Noulet, F., Walter, M.C., Morandi, L., Poupiot, J., Roudaut, C., Bittner, R.E., Bartoli, M. and Richard, I.: « A third of LGMD2A biopsies have normal calpain 3 proteolytic activity as determined by an in vitro assay » Neuromuscul Disord 17 (2007) 148-56.
Paldi, A.: « Expression stochastique des gènes » Génétiquement indéterminé: le vivant auto-organisé. Editions QUAE – INRA, 2007.
Prongidi-Fix, L., Sugawara, M., Bertani, P., Raya, J., Leborgne, C., Kichler, A. and Bechinger, B.: « Self-Promoted Cellular Uptake of Peptide/DNA Transfection Complexes » Biochemistry 46 (2007) 11253-62.
Quenneville, S.P., Chapdelaine, P., Skuk, D., Paradis, M., Goulet, M., Rousseau, J., Xiao, X.,Garcia, L. and Tremblay, J.P.: « Autologous transplantation of muscle precursor cells modified with a lentivirus for muscular dystrophy: human cells and primate models » Mol Ther 15 (2007) 431-8.
Rivière, C. and Douar, A.M.: « Current challenges in AAV systemic delivery in humans. in Virus Expression Vectors » In: Hefferon, K.L. (Ed.), Virus expression vectors. Transworld Research Network, 2007, pp. Chapt 2.
Robin, P., Fritsch, L., Philipot, O., Svinartchouk, F. and Ait-Si-Ali, S.: « Post-translational modifications of histones H3 and H4 associated with the histone methyltransferases Suv39h1 and G9a » Genome Biol 8 (2007) R270.
Stevanin, G., Paternotte, C., Coutinho, P., Klebe, S., Elleuch, N., Loureiro, J.L., Denis, E., Cruz, V.T., Durr, A., Prud'homme, J.F., Weissenbach, J., Brice, A. and Hazan, J.: « A new locus for autosomal recessive spastic paraplegia (SPG32) on chromosome 14q12-q21 » Neurology 68 (2007) 1837-40.
Stevanin, G., Santorelli, F.M., Azzedine, H., Coutinho, P., Chomilier, J., Denora, P.S., Martin, E., Ouvrard-Hernandez, A.M., Tessa, A., Bouslam, N., Lossos, A., Charles, P., Loureiro, J.L., Elleuch, N., Confavreux, C., Cruz, V.T., Ruberg, M., Leguern, E., Grid, D., Tazir, M., Fontaine, B., Filla, A., Bertini, E., Durr, A. and Brice, A.: « Mutations in SPG11, encoding spatacsin, are a major cause of spastic paraplegia with thin corpus callosum » Nat Genet 39 (2007) 366-72.
Stockholm, D., Benchaouir, R., Picot, J., Rameau, P., Neildez, T.M.A., Landini, G., Laplace-Builhe, C. and Paldi, A.: « The origin of phenotypic heterogeneity in a clonal cell population in vitro » PLoS ONE 2 (2007) e394.
Tang, Y., Liu, X., Zoltoski, R.K., Novak, L.A., Herrera, R.A., Richard, I., Kuszak, J.R. and Kumar, N.M.: « Age-Related Cataracts in {alpha}3Cx46-Knockout Mice Are Dependent on a Calpain 3 Isoform » Invest Ophthalmol Vis Sci 48 (2007) 2685-94.
Vallat, J.M., Magy, L., Lagrange, E., Sturtz, F., Magdelaine, C., Grid, D. and Tazir, M.: « Diagnostic value of ultrastructural nerve examination in Charcot-Marie-Tooth disease: two CMT 1B cases with pseudo-recessive inheritance » Acta Neuropathol (Berl) 113 (2007) 443-9.
Vassilopoulos, S., Brocard, J., Garcia, L., Marty, I. and Bouron, A.: « Retrograde regulation of store-operated calcium channels by the ryanodine receptor-associated protein triadin 95 in rat skeletal myotubes » Cell Calcium 41 (2007) 179-185.
Veron, P., Allo, V., Riviere, C., Bernard, J., Douar, A.M. and Masurier, C.: « Major subsets of human dendritic cells are efficiently transduced using self-complementary adeno-associated viral vectors 1 and 2 » J Virol 81 (2007) 5385-5394.

2006

Aranyi, T. and Paldi, A.: « The constant variation: DNA methylation changes during preimplantation development » FEBS Lett 580 (2006) 6521-6.
Azzedine, H., Ravise, N., Verny, C., Gabreels-Festen, A., Lammens, M., Grid, D., Vallat, J.M., Durosier, G., Senderek, J., Nouioua, S., Hamadouche, T., Bouhouche, A., Guilbot, A., Stendel, C., Ruberg, M., Brice, A., Birouk, N., Dubourg, O., Tazir, M. and LeGuern, E.: « Spine deformities in Charcot-Marie-Tooth 4C caused by SH3TC2 gene mutations » Neurology 67 (2006) 602-6.
Balci, B., Aurino, S., Haliloglu, G., Talim, B., Erdem, S., Akcoren, Z., Tan, E., Caglar, M., Richard, I., Nigro, V., Topaloglu, H. and Dincer, P.: « Calpain-3 mutations in Turkey » Eur J Pediatr 165 (2006) 293-8.
Barde, I., Zanta-Boussif, M.A., Paisant, S., Leboeuf, M., Rameau, P., Delenda, C. and Danos, O.: « Efficient control of gene expression in the hematopoietic system using a single Tet-on inducible lentiviral vector » Mol Ther 13 (2006) 382-90.
Bartoli, M., Bourg, N., Stockholm, D., Raynaud, F., Delevaque, A., Han, Y., Borel, P., Seddik, K., Armande, N. and Richard, I.: « A mice model for monitoring calpain activity in physiological and pathological conditions » J Biol Chem 281 (2006) 39672-80.
Bartoli, M., Roudaut, C., Martin, S., Fougerousse, F., Suel, L., Poupiot, J., Gicquel, E., Noulet, F., Danos, O. and Richard, I.: « Safety and Efficacy of AAV-Mediated Calpain 3 Gene Transfer in a Mouse Model of Limb-Girdle Muscular Dystrophy Type 2A » Mol Ther 13 (2006) 250-259.
Beroud, C., Tuffery-Giraud, S., Matsuo, M., Hamroun, D., Humbertclaude, V., Monnier, N., Moizard, M.P., Voelckel, M.A., Calemard, L.M., Boisseau, P., Blayau, M., Philippe, C., Cossee, M., Pages, M., Rivier, F., Danos, O., Garcia, L. and Claustres, M.: « Multiexon skipping leading to an artificial DMD protein lacking amino acids from exons 45 through 55 could rescue up to 63% of patients with Duchenne muscular dystrophy » Hum Mutat 28 (2006) 196 – 202.
Blewitt, M.E., Vickaryous, N.K., Paldi, A., Koseki, H. and Whitelaw, E.: « Dynamic reprogramming of DNA methylation at an epigenetically sensitive allele in mice » PLoS Genet 2 (2006) e49.
Brissault, B., Kichler, A., Leborgne, C., Danos, O., Cheradame, H., Gau, J., Auvray, L. and Guis, C.: « Synthesis, characterization, and gene transfer application of poly(ethylene glycol-b-ethylenimine) with high molar mass polyamine block. » Biomacromolecules 7 (2006) 2863-2870.
Brissault, B., Leborgne, C., Guis, C., Danos, O., Cheradame, H. and Kichler, A.: « Linear topology confers in vivo gene transfer activity to polyethylenimines » Bioconjug Chem 17 (2006) 759-65.
Burckbuchler, V., Wintgens, V., Lecomte, S., Percot, A., Leborgne, C., Danos, O., Kichler, A.and Amiel, C.: « DNA compaction into new DNA vectors based on cyclodextrin polymer: SERS characterization » Biopolymers 81 (2006) 360-70.
Cabarrocas, J., Cassan, C., Magnusson, F., Piaggio, E., Mars, L., Derbinski, J., Kyewski, B.,Gross, D.A., Salomon, B.L., Khazaie, K., Saoudi, A. and Liblau, R.S.: « Foxp3+ CD25+ regulatory T cells specific for a neo-self-antigen develop at the double-positive thymic stage » Proc Natl Acad Sci U S A 103 (2006) 8453-8.
Capron, C., Lecluse, Y., Kaushik, A.L., Foudi, A., Lacout, C., Sekkai, D., Godin, I., Albagli, O., Poullion, I., Svinartchouk, F., Schanze, E., Vainchenker, W., Sablitzky, F., Bennaceur-Griscelli, A. and Dumenil, D.: « The SCL relative LYL-1 is required for fetal and adult hematopoietic stem cell function and B-cell differentiation » Blood 107 (2006) 4678-86.
Carmo, M., Faria, T.Q., Falk, H., Coroadinha, A.S., Teixeira, M., Merten, O.-W., Gény-Fiamma, C., Alves, P.M., Danos, O., Panet, A., Carrondo, M.J.T. and Cruz, P.E.: « Relationship between retroviral vector membrane and vector stability » J Gen Virol 87 (2006) 1349-56.
Cheradame, H., Brissault, B., Guis, C., Guégan, P., Pomel, C., Kichler, A., Gau, J. and Auvray, L.: « Improvement of synthetic vectors for gene therapy using ring-opening cationic polymerization » Macromolecular Symposia 240 (2006) 166-177.
Coroadinha, A.S., Alves, P.M., Santos, S.S., Cruz, P.E., Merten, O.-W. and Carrondo, M.J.: « Retrovirus producer cell line metabolism: implications on viral productivity » Appl Microbiol Biotechnol 72 (2006) 1125-35.
Coroadinha, A.S., Ribeiro, J., Roldao, A., Cruz, P.E., Alves, P.M., Merten, O.-W. and Carrondo, M.J.: « Effect of medium sugar source on the production of retroviral vectors for gene therapy » Biotechnol Bioeng 94 (2006) 24-36.
Danos, O. and Svinartchouk, F.: « Dialysis-assisted two-dimensional gel electrophoresis » Electrophoresis 27 (2006) 3475 – 3479.
Deburgrave, N., Daoud, F., Llense, S., Barbot, J.C., Recan, D., Peccate, C., Burghes, A.H., Beroud, C., Garcia, L., Kaplan, J.C., Chelly, J. and Leturcq, F.: « Protein- and mRNA-based phenotype-genotype correlations in DMD/BMD with point mutations and molecular basis for BMD with nonsense and frameshift mutations in the DMD gene » Hum Mutat 28 (2006) 183 – 195.
Dubourg, O., Azzedine, H., Verny, C., Durosier, G., Birouk, N., Gouider, R., Salih, M., Bouhouche, A., Thiam, A., Grid, D., Mayer, M., Ruberg, M., Tazir, M., Brice, A. and LeGuern, E.: « Autosomal-recessive forms of demyelinating Charcot-Marie-Tooth disease » Neuromolecular Med 8 (2006) 75-86.
Duguez, S., Bartoli, M. and Richard, I.: « Calpain 3: a key regulator of the sarcomere? » Febs J 273 (2006) 3427-36.
Elleuch, N., Depienne, C., Benomar, A., Hernandez, A.M., Ferrer, X., Fontaine, B., Grid, D., Tallaksen, C.M., Zemmouri, R., Stevanin, G., Durr, A. and Brice, A.: « Mutation analysis of the paraplegin gene (SPG7) in patients with hereditary spastic paraplegia » Neurology 66 (2006) 654-9.
Etzel, C.J., Chen, W.V., Shepard, N., Jawaheer, D., Cornelis, F., Seldin, M.F., Gregersen, P.K. and Amos, C.I.: « Genome-wide meta-analysis for rheumatoid arthritis » Human Genet 119 (2006) 634-641.
Gross, D.A., Chappert, P., Leboeuf, M., Monteilhet, V., Van Wittenberghe, L., Danos, O. and Davoust, J.: « Simple conditioning with mono-specific CD4+CD25+ regulatory T cells for bone marrow engraftment and tolerance to multiple gene products » Blood 108 (2006) 1841-1848.
Hempelmann, A., Taylor, K.P., Heils, A., Lorenz, S., Prud'homme, J.F., Nabbout, R., Dulac, O., Rudolf, G., Zara, F., Bianchi, A., Robinson, R., Gardiner, R.M., Covanis, A., Lindhout, D., Stephani, U., Elger, C.E., Weber, Y.G., Lerche, H., Nurnberg, P., Kron, K.L., Scheffer, I.E., Mulley, J.C., Berkovic, S.F. and Sander, T.: « Exploration of the genetic architecture of idiopathic generalized epilepsies » Epilepsia 47 (2006) 1682-90.
Izmiryan, A., Cheraud, Y., Khanamiryan, L., Leterrier, J.F., Federici, T., Peltekian, E., Moura-Neto, V., Paulin, D., Li, Z. and Xue, Z.G.: « Different expression of synemin isoforms in glia and neurons during nervous system development » Glia 54 (2006) 204-213.
Kichler, A., Mason, A.J. and Bechinger, B.: « Cationic amphipathic histidine-rich peptides for gene delivery » Biochim Biophys Acta 1758 (2006) 301-7.
Krahn, M., Bernard, R., Pecheux, C., Hammouda, E.H., Eymard, B., De Munain, A.L., Cobo, A.M., Romero, N., Urtizberea, A., Leturcq, F. and Levy, N.: « Screening of the CAPN3 gene in patients with possible LGMD2A » Clin Genet 69 (2006) 444-449.
Lagresle-Peyrou, C., Yates, F., Malassis-Seris, M., Hue, C., Morillon, E., Garrigue, A., Liu, A., Hajdari, P., Stockholm, D., Danos, O., Lemercier, B., Gougeon, M.L., Rieux-Laucat, F., de Villartay, J.P., Fischer, A. and Cavazzana-Calvo, M.: « Long term immune reconstitution in RAG-1 deficient mice treated by retroviral gene therapy: a balance between efficiency and toxicity » Blood 107 (2006) 63-72.
Lefevre, C., Bouadjar, B., Ferrand, V., Tadini, G., Megarbane, A., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Mutations in a new cytochrome P450 gene in lamellar ichthyosis type 3 » Hum Mol Genet 15 (2006) 767-76.
Mantovani, J., Holic, N., Martinez, K., Danos, O. and Perea, J.: « A high throughput method for genome-wide analysis of retroviral integration » Nucleic Acids Res 34 (2006) e134.
Marquis, J., Paillard, L., Audic, Y., Cosson, B., Danos, O., Le Bec, C. and Osborne, H.B.: « CUG-BP1/CELF1 requires UGU-rich sequences for high affinity binding » Biochem J 400 (2006) 291-301.
Mas, A., Montane, J., Anguela, X.M., Munoz, S., Douar, A.M., Riu, E., Otaegui, P. and Bosch, F.: « Reversal of type 1 diabetes by engineering a glucose sensor in skeletal muscle » Diabetes 55 (2006) 1546-53.
Mason, A.J., Gasnier, C., Kichler, A., Prévost, G., Aunis, D., Metz-Boutigue, M.-H. and Bechinger, B.: « Designed histidine-rich peptides show pH dependent antibiotic action against pathogenic bacteria » Antimicrob Agents Chemother. 2006 (2006) 3305-3311.
Mason, A.J., Martinez, A., Glaubitz, C., Danos, O., Kichler, A. and Bechinger, B.: « The antibiotic and DNA transfecting peptide LAH4 selectively associates with, and disorders, anionic lipids in mixed membranes » FASEB Journal 20 (2006) 320-2.
Olivier, A., Jeanson-Leh, L., Bouma, G., Compagno, D., Blondeau, J., Seye, K., Charrier, S., Burns, S., Thrasher, A., Danos, O., Vainchenker, W. and Galy, A.: « A partial down-regulation of WASP is sufficient to inhibit podosome formation in dendritic cells » Mol Ther 13 (2006) 729-37.
Olivier, A., Lauret, E., Gonin, P. and Galy, A.: « The Notch ligand delta-1 is a hematopoietic development co-factor for plasmacytoid dendritic cells » Blood 107 (2006) 2694-701.
Paldi, A.: « Empreinte génomique » Métabolismes Hormones Diabètes et Nutrition 10 (2006) 6-9.
Parouchev, A., Nguyen, T.H., Dagher, I., Mainot, S., Groyer-Picard, M.T., Branger, J., Gonin, P., Di Santo, J., Franco, D., Gras, G. and Weber, A.: « Efficient ex vivo gene transfer into non-human primate hepatocytes using HIV-1 derived lentiviral vectors » J Hepatol 45 (2006) 99-107.
Partridge, T.: « Disciplining the stem cell into myogenesis » N Engl J Med 354 (2006) 1844-1845.
Rameau, P. and Laplace-Builhe, C.: « Le tri à haute vitesse » In: Ronot, X., Grunwald, D., Mayol, J.F. and Boutonnat, J. (Eds.), La cytométrie en flux: un besoin circonstanciel en biologie et en médecine. Technique &amp; Documentation, Paris, 2006.
Richard, I.: « Limb-girdle muscular dystrophies » In: Ganten, D.R., K. (Ed.), Encyclopedic Reference of Genomics and Proteomics. Springer-Verlag, 2006.
Rowe, H.M., Lopes, L., Ikeda, Y., Bailey, R., Barde, I., Zenke, M., Chain, B.M. and Collins, M.K.: « Immunization with a Lentiviral Vector Stimulates both CD4 and CD8 T Cell Responses to an Ovalbumin Transgene » Mol Ther 13 (2006) 310-9.
Schucht, R., Coroadinha, A.S., Zanta-Boussif, M.A., Verhoeyen, E., Carrondo, M.J., Hauser, H. and Wirth, D.: « A new generation of retroviral producer cells: predictable and stable virus production by flp-mediated site-specific integration of retroviral vectors » Mol Ther 14 (2006) 285-92.
Smet-Nocca, C., Paldi, A. and Benecke, A.: « De l'épigénomique à l'émergence morphogénétique » In: Bourgine, P. and Lesne, A. (Eds.), Morphogenèse. L'origine des formes. Belin, Paris, 2006.
Spirito, F., Capt, A., Del Rio, M., Larcher, F., Guaguere, E., Danos, O. and Meneguzzi, G.: « Sustained phenotypic reversion of junctional epidermolysis bullosa dog keratinocytes: Establishment of an immunocompetent animal model for cutaneous gene therapy » Biochem Biophys Res Commun 339 (2006) 769-778.
Striano, P., Lispi, M.L., Gennaro, E., Madia, F., Traverso, M., Bordo, L., Aridon, P., Boneschi, F.M., Barone, B., dalla Bernardina, B., Bianchi, A., Capovilla, G., De Marco, P., Dulac, O., Gaggero, R., Gambardella, A., Nabbout, R., Prud'homme, J.F., Day, R., Vanadia, F., Vecchi, M., Veggiotti, P., Vigevano, F., Viri, M., Minetti, C. and Zara, F.: « Linkage analysis and disease models in benign familial infantile seizures: A study of 16 families » Epilepsia 47 (2006) 1029-1034.
Striano, P., Lispi, M.L., Gennaro, E., Madia, F., Traverso, M., Bordo, L., Aridon, P., Boneschi, F.M., Barone, B., dalla Bernardina, B., Bianchi, A., Capovilla, G., De Marco, P., Dulac, O., Gaggero, R., Gambardella, A., Nabbout, R., Prud'homme, J.F., Day, R., Vanadia, F., Vecchi, M., Veggiotti, P., Vigevano, F., Viri, M., Minetti, C. and Zara, F.: « Linkage analysis and disease models in benign familial infantile seizures: a study of 16 families » Epilepsia 47 (2006) 1029-34.
t'Hoen, P.A., van der Wees, C.G., Aartsma-Rus, A., Turk, R., Goyenvalle, A., Danos, O.,Garcia, L., van Ommen, G.J., den Dunnen, J.T. and van Deutekom, J.C.: « Gene expression profiling to monitor therapeutic and adverse effects of antisense therapies for Duchenne muscular dystrophy » Pharmacogenomics 7 (2006) 281-97.
Veron, P., Boutin, S., Bernard, J., Danos, O., Davoust, J. and Masurier, C.: « Efficient transduction of monocyte- and CD34(+)- derived Langerhans cells with lentiviral vectors in the absence of phenotypic and functional maturation » J Gene Med 8 (2006) 951 – 961.
Warnock, J.N., Merten, O.-W. and Al, R.M.: « Cell culture processes for the production of viral vectors for gene therapy purposes » Cytotechnology 50 (2006) 141.

2005

Abecasis, G., Allen, M., Barker, J., Burden, D., Capon, F., Christophers, E., Elder, J.T., Fischer, J., Gudjonsson, J.E., Huffmeier, U., Jenisch, S., Karason, A., Kere, J., Nair, R.P., Novelli, G., Prud'homme, J.F., Qin, Z.H.S., Samuelsson, L., Sanchez, F., Saarialho-Kere, U., Stahle, M., Stuart, P., Tillman, D., Traupe, H., Trembath, R., Valdimarsson, H., Veal, C., Voorhees, J.J. and Weichenthal, M.: « Fine mapping of the psoriasis susceptibility gene PSORS1: A reassessment of risk associated with a putative risk haplotype lacking HLA-Cw6 » J Invest Dermatol 124 (2005) 921-930.
Apparailly, F., Khoury, M., Vervoordeldonk, M.J.B., Adriaansen, J., Gicquel, E., Perez, N., Riviere, C., Louis-Plence, P., Noel, D., Danos, O., Douar, A.M., Tak, P.P. and Jorgensen, C.: « Adeno-associated virus pseudotype 5 vector improves gene transfer in arthritic joints » Hum Gene Ther 16 (2005) 426-434.
Baconnais, S., Delavoie, F., Zahm, J.M., Milliot, M., Terryn, C., Castillon, N., Banchet, V., Michel, J., Danos, O., Merten, M., Chinet, T., Zierold, K., Bonnet, N., Puchelle, E. and Balossier, G.: « Abnormal ion content, hydration and granule expansion of the secretory granules from cystic fibrosis airway glandular cells » Exp Cell Res 309 (2005) 296-304.
Bagnis, C., Merten, O.-W. and Mezzina, M.: « Advanced methods for industrial production, purification, and characterization of gene vectors » Gene Ther 12 (2005) 1-177.
Bartoli, M., Poupiot, J., Goyenvalle, A., Perez, N., Garcia, L., Danos, O. and Richard, I.: « Noninvasive monitoring of therapeutic gene transfer in animal models of muscular dystrophies » Gene Ther 13 (2005) 20-28.
Bartoli, M. and Richard, I.: « Calpains and muscle wasting » Int J Biochem Cell Biol 37 (2005) 2115-2133.
Berthebaud, M., Riviere, C., Jarrier, P., Foudi, A., Zhang, Y., Compagno, D., Galy, A., Vainchenker, W. and Louache, F.: « RGS16 is a negative regulator of SDF-1-CXCR4 signaling in megakaryocytes » Blood 106 (2005) 2962-8.
Borgnon, J., Djamouri, F., Lorand, I., Di Rico, V., Loux, N., Pages, J.C., Franco, D., Capron, F. and Weber, A.: « Follistatin allows efficient retroviral-mediated gene transfer into rat liver » Biochem Biophys Res Commun 328 (2005) 937-943.
Charrier, S., Stockholm, D., Seye, K., Opolon, P., Taveau, M., Gross, D.A., Bucher-Laurent, S., Delenda, C., Vainchenker, W., Danos, O. and Galy, A.: « A lentiviral vector encoding the human Wiskott-Aldrich syndrome protein corrects immune and cytoskeletal defects in WASP knockout mice » Gene Ther 12 (2005) 597-606.
Coecke, S., Balls, M., Bowe, G., Davis, J., Gstraunthaler, G., Hartung, T., Hay, R., Merten, O.-W., Price, A., Schechtman, L., Stacey, G. and Stokes, W.: « Guidance on Good Cell Culture Practice: A Report of the Second ECVAM Task Force on Good Cell Culture Practice » Alternatives to laboratory animals: ATLA. 33 (2005) 261-287.
Collins, C.A. and Partridge, T.A.: « Self-renewal of the adult skeletal muscle satellite cell » Cell Cycle 4 (2005) 1338-1341.
Cruz, P.E., Carmo, M., Coroadinha, A.S., Bengala, A., Goncalves, D., Teixeira, M., Merten, O.-W., Gény-Fiamma, C. and Carrondo, M.J.T.: « Retroviral vector stability: inactivation kinetics and membrane properties. » In: Godia, F. and Fussenegger, M. (Eds.), Animal cell technology meets genomics. Springer, Dordrecht, 2005, pp. 303-308.
Davoust, J., Bertho, N., Masurier, C. and Gross, D.: « Immunity and tolerance induction in gene therapy » In: Dunn, M.J., Jorde, L.B., Little, P.F.R. and Subramaniam, S. (Eds.), Encyclopedia of genetics, genomics, proteomics and bioinformatics. John Wiley &amp; Sons, Ltd., New York, 2005, pp. 817-25.
De Sandre-Giovannoli, A., Delague, V., Hamadouche, T., Chaouch, M., Krahn, M., Boccaccio, I., Maisonobe, T., Chouery, E., Jabbour, R., Atweh, S., Grid, D., Megarbane, A. and Levy, N.: « Homozygosity mapping of autosomal recessive demyelinating Charcot-Marie-Tooth neuropathy (CMT4H) to a novel locus on chromosome 12p11.21-q13.11 » J Med Genet 42 (2005) 260-5.
Decraene, C., Benchaouir, R., Dillies, M.A., Israeli, D., Bortoli, S., Rochon, C., Rameau, P., Pitaval, A., Tronik-Le Roux, D., Danos, O., Gidrol, X., Garcia, L. and Pietu, G.: « Global transcriptional characterization of SP and MP cells from the myogenic C2C12 cell line: effect of FGF6 » Physiol Genomics 23 (2005) 132-49.
Decraene, C., Benchaouir, R., Dillies, M.A., Israeli, D., Bortoli, S., Rochon, C., Rameau, P., Pitaval, A., Tronik-Le Roux, D., Danos, O., Gidrol, X., Garcia, L. and Pietu, G.: « Global transcriptional characterization of SP and MP cells from the myogenic C2C12 cell line: effect of FGF6 » Physiological Genomics 23 (2005) 132-149.
Delenda, C. and Gaillard, C.: « Real-time quantitative PCR for the design of lentiviral vector analytical assays » Gene Ther 12 Suppl 1 (2005) S36-50.
Fleurence, E., Riviere, C., Lacaze-Masmonteil, T., Franco-Motoya, M.L., Waszak, P., Bourbon, J., Danos, O., Douar, A.M. and Delacourt, C.: « Comparative efficacy of intratracheal adeno-associated virus administration to newborn rats » Hum Gene Ther 16 (2005) 1298-306.
Gagnoux-Palacios, L., Hervouet, C., Spirito, F., Roques, S., Mezzina, M., Danos, O. and Meneguzzi, G.: « Assessment of optimal transduction of primary human skin keratinocytes by viral vectors » J Gene Med 7 (2005) 1178-86.
Gonin, P.: « Entamoeba histolytica, Entamoeba dispar » In: Fuchs, J. and Podda, M. (Eds.), Encyclopedia of Medical Genomics and Proteomics. Marcel Dekker, New-York, 2005, pp. 391 – 394.
Gonin, P., Arandel, L., Van Wittenberghe, L., Marais, T., Perez, N. and Danos, O.: « Femoral intra-arterial injection: a tool to deliver and assess recombinant AAV constructs in rodents whole hind limb » J Gene Med 7 (2005) 782-91.
Gonin, P., Buchholz, C.J., Pallardy, M. and Mezzina, M.: « Gene therapy bio-safety: scientific and regulatory issues » Gene Ther 12 Suppl 1 (2005) S146-52.
Imamura, T., Kerjean, A., Heams, T., Kupiec, J.J., Thenevin, C. and Paldi, A.: « Dynamic CpG and non-CpG methylation of the Peg1/Mest gene in the mouse oocyte and preimplantation embryo » J Biol Chem 280 (2005) 20171-5.
Jenny, C., Toublanc, E., Danos, O. and Merten, O.-W.: « Evaluation of a Serum-free Medium for the Production of rAAV-2 using HeLa Derived Producer Cells » Cytotech 49 (2005) 11-23.
Kichler, A., Leborgne, C., Savage, P.B. and Danos, O.: « Cationic steroid antibiotics demonstrate DNA delivery properties » J Control Release 107 (2005) 174-82.
Kissenpfennig, A., Henri, S., Dubois, B., Laplace-Builhe, C., Perrin, P., Romani, N., Tripp, C.H., Douillard, P., Leserman, L., Kaiserlian, D., Saeland, S., Davoust, J. and Malissen, B.: « Dynamics and function of Langerhans cells in vivo dermal dendritic cells colonize lymph node areas distinct from slower migrating Langerhans cells » Immunity 22 (2005) 643-54.
Kocaefe, Y.C., Israeli, D., Ozguc, M., Danos, O. and Garcia, L.: « Myogenic program induction in mature fat tissue (with MyoD expression) » Exp Cell Res 308 (2005) 300-8.
Komura, E., Tonetti, C., Penard-Lacronique, V., Chagraoui, H., Lacout, C., Lecouedic, J.P., Rameau, P., Debili, N., Vainchenker, W. and Giraudier, S.: « Role for the nuclear factor kappaB pathway in transforming growth factor-beta1 production in idiopathic myelofibrosis: possible relationship with FK506 binding protein 51 overexpression » Cancer Res 65 (2005) 3281-9.
Kreij, K., Mandenius, C.F., Clemente, J.J., Cunha, A.E., Monteiro, S.M.S., Carrondo, M.J.T., Hesse, F., Bassani Molinas, M.d.l.M., Wagner, R., Merten, O.-W., Gény-Fiamma, C., Leger, W., Wiesinger-Mayr, H., Müller, D., Katinger, H., Martensson, P., Bachinger, T. and Mitrovics, J.: « On-line detection of microbial contaminations in animal cell reactor cultures using an electronic nose device » Cytotech 48 (2005) 41-58.
Kreij, K., Mandenius, C.F., Clemente, J.J., Cunha, A.E., Monteiro, S.M.S., Carrondo, M.J.T., Hesse, F., Molinas, M., Wagner, R., Merten, O.W., Geny-Katinger, C., Martensson, P., Bachinger, T. and Mitrovics, J.: « On-line detection of microbial contaminations in animal cell reactor cultures using an electronic nose device » Cytotechnology 48 (2005) 41-58.
Lange, S., Xiang, F., Yakovenko, A., Vihola, A., Hackman, P., Rostkova, E., Kristensen, J., Brandmeier, B., Franzen, G., Hedberg, B., Gunnarsson, L.G., Hughes, S.M., Marchand, S., Sejersen, T., Richard, I., Edstrom, L., Ehler, E., Udd, B. and Gautel, M.: « The kinase domain of titin controls muscle gene expression and protein turnover » Science 308 (2005) 1599-603.
Merten, O.-W., Gény-Fiamma, C. and Douar, A.M.: « Current issues in adeno-associated viral vectors production » Gene Ther 12 (2005) S51-S61.
Pascolo, S., Ginhoux, F., Laham, N., Walter, S., Schoor, O., Probst, J., Rohrlich, P., Obermayr, F., Fisch, P., Danos, O., Ehrlich, R., Lemonnier, F.A. and Rammensee, H.G.: « The non-classical HLA class I molecule HFE does not influence the NK-like activity contained in fresh human PBMCs and does not interact with NK cells » Int Immunol 17 (2005) 117-122.
Proust, A., Guillet, B., Pellier, I., Rachieru, P., Hoarau, C., Claeyssens, S., Leonard, C., Charrier, S., Vainchenker, W., Tchernia, G. and Delaunay, J.: « Recurrent V75M mutation within the Wiskott-Aldrich syndrome protein: description of a homozygous female patient » Eur J Haematol 75 (2005) 54-9.
Rezgui, S.S., Vassilopoulos, S., Brocard, J., Platel, J.C., Bouron, A., Arnoult, C., Oddoux, S., Garcia, L., De Waard, M. and Marty, I.: « Triadin (trisk 95) overexpression blocks excitation-contraction coupling in rat skeletal myotubes » J Biol Chem 280 (2005) 39302-8.
Rohrlich, P.S., Fazilleau, N., Ginhoux, F., Firat, H., Michel, F., Cochet, M., Laham, N., Roth, M.P., Pascolo, S., Nato, F., Coppin, H., Charneau, P., Danos, O., Acuto, O., Ehrlich, R., Kanellopoulos, J. and Lemonnier, F.A.: « Direct recognition by alphabeta cytolytic T cells of Hfe, a MHC class Ib molecule without antigen-presenting function » Proc Natl Acad Sci U S A 102 (2005) 12855-60.
Stockholm, D., Bartoli, M., Sillon, G., Bourg, N., Davoust, J. and Richard, I.: « Imaging calpain protease activity by multiphoton FRET in living mice » J Mol Biol 346 (2005) 215-222.
Tine, J.A., Firat, H., Payne, A., Russo, G., Davis, S.W., Tartaglia, J., Lemonnier, F.A., Demoyen, P.L. and Moingeon, P.: « Enhanced multiepitope-based vaccines elicit CD8(+) cytotoxic T cells against both immunodominant and cryptic epitopes » Vaccine 23 (2005) 1085-91.
Udd, B., Vihola, A., Sarparanta, J., Richard, I. and Hackman, P.: « Titinopathies and extension of the M-line mutation phenotype beyond distal myopathy and LGMD2J » Neurology 64 (2005) 636-42.
Vallat, J.M., Grid, D., Magdelaine, C., Sturtz, F., Levy, N. and Tazir, M.: « [Autosomal recessive forms of Charcot-Marie-Tooth disease] » Bull Acad Natl Med 189 (2005) 55-68; discussion 68-9.
Vallat, J.M., Tazir, M., Magdelaine, C., Sturtz, F. and Grid, D.: « Autosomal-recessive Charcot-Marie-Tooth diseases » J Neuropathol Exp Neurol 64 (2005) 363-70.
Vendome, J., Letard, S., Martin, F., Svinarchuk, F., Dubreuil, P., Auclair, C. and Le Bret, M.: « Molecular modeling of wild-type and D816V c-Kit inhibition based on ATP-competitive binding of ellipticine derivatives to tyrosine kinases » J Med Chem 48 (2005) 6194-201.
Young, J., Morbois-Trabut, L., Couzinet, B., Lascols, O., Dion, E., Béréziat, V., Fève, B., Richard, I., Capeau, J., Chanson, P. and Vigouroux, C.: « Type A insulin resistance syndrome revealing a novel lamin A mutation » Diabetes 54 (2005) 1873-1878.

2004

Beauchamp-Nicoud, A., Da Costa, L., Proust, A., Rince, P., Saker, S. and Tchernia, G.: « Postmortem diagnosis of Diamond-Blackfan anemia » J Pediatr Hematol Oncol 26 (2004) 847-8.
Benchaouir, R., Rameau, P., Decraene, C., Dreyfus, P., Israeli, D., Pietu, G., Danos, O. and Garcia, L.: « Evidence for a resident subset of cells with SP phenotype in the C2C12 myogenic line: a tool to explore muscle stem cell biology » Exp Cell Res 294 (2004) 254-68.
Bidou, L., Hatin, I., Perez, N., Allamand, V., Panthier, J.J. and Rousset, J.P.: « Premature stop codons involved in muscular dystrophies show a broad spectrum of readthrough efficiencies in response to gentamicin treatment » Gene Ther 11 (2004) 619-27.
Borg, C., Abdelali, J., Laderach, D., Maruyama, K., Wakasugi, H., Charrier, S., Ryffel, B., Cambi, A., Figdor, C., Vainchenker, W., Galy, A., Caignard, A. and Zitvogel, L.: « NK cell activation by dendritic cells (DCs) requires the formation of a synapse leading to IL-12 polarization in DCs » Blood 104 (2004) 3267-3275.
Castillon, N., Avril-Delplanque, A., Coraux, C., Delenda, C., Peault, B., Danos, O. and Puchelle, E.: « Regeneration of a well-differentiated human airway surface epithelium by spheroid and lentivirus vector-transduced airway cells » J Gene Med 6 (2004) 846-56.
Caux, F., Selma, Z.B., Laroche, L., Prud'homme, J.F. and Fischer, J.: « CGI-58/ABHD5 gene is mutated in Dorfman-Chanarin syndrome » Am J Med Genet A 129 (2004) 214.
Delaunay, J., Nouyrigat, V., Proust, A., Schischmanoff, P.O., Cynober, T., Yvart, J., Gaillard, C., Danos, O. and Tchernia, G.: « Different impacts of alleles alphaLEPRA and alphaLELY as assessed versus a novel, virtually null allele of the SPTA1 gene in trans » Br J Haematol 127 (2004) 118-22.
Delaunay, J., Nouyrigat, V., Proust, A., Schischmanoff, P.O., Cynober, T., Yvart, J., Gaillard, C., Danos, O. and Tchernia, G.: « Different impacts of alleles alpha(LEPRA) and alpha(LELY) as assessed versus a novel, virtually null allele of the SPTA1 gene in trans » Br J Haematol 127 (2004) 118-122.
Demir, E., Prud'homme, J.F. and Topcu, M.: « Infantile convulsions and paroxysmal choreoathetosis in a consanguineous family » Pediatr Neurol 30 (2004) 349-53.
Dreyfus, P.A., Chretien, F., Chazaud, B., Kirova, Y., Caramelle, P., Garcia, L., Butler-Browne, G. and Gherardi, R.K.: « Adult bone marrow-derived stem cells in muscle connective tissue and satellite cell niches » Am J Pathol 164 (2004) 773-9.
Duan, M., Venail, F., Spencer, N. and Mezzina, M.: « Treatment of peripheral sensorineural hearing loss: gene therapy » Gene Ther 11 (2004) S51-S56.
Gény-Fiamma, C., Millot, L., Rocca, C., Danos, O. and Merten, O.-W.: « Optimization of the production of retroviral vectors – Influences of the sugar source » In: Kitagawa, Y., Matsuda, T. and Iijima, S. (Eds.), Animal cell technology: basic and applied aspects. Kluwer Academic Publishers, Dordrecht, 2004, pp. 89-97.
Ginhoux, F., Turbant, S., Gross, D.A., Poupiot, J., Marais, T., Lone, Y., Lemonnier, F.A., Firat, H., Perez, N., Danos, O. and Davoust, J.: « HLA-A*0201-restricted cytolytic responses to the rtTA transactivator dominant and cryptic epitopes compromise transgene expression induced by the tetracycline on system » Mol Ther 10 (2004) 279-89.
Goyenvalle, A., Vulin, A., Fougerousse, F., Leturcq, F., Kaplan, J.C., Garcia, L. and Danos, O.: « [An opening in Duchenne muscular dystrophy: persistent therapeutic rescue of dystrophin by vectorized antisense mediated exon skipping in mdx mice.] » Med Sci (Paris) 20 (2004) 1163-1165.
Goyenvalle, A., Vulin, A., Fougerousse, F., Leturcq, F., Kaplan, J.C., Garcia, L. and Danos, O.: « Rescue of dystrophic muscle through U7 snRNA-mediated Exon-Skipping » Science 306 (2004) 1796-9.
Gross, D.A., Graff-Dubois, S., Opolon, P., Cornet, S.S., Alves, P., Bennaceur-Griscelli, A., Faure, O., Guillaume, P., Firat, H.H., Chouaib, S., Lemonnier, F.A., Davoust, J., Miconnet, I., Vonderheide, R.H. and Kosmatopoulos, K.: « High vaccination efficiency of low-affinity epitopes in antitumor immunotherapy » J Clin Invest 113 (2004) 425-33.
Haack, K., Cockrell, A.S., Ma, H., Israeli, D., Ho, S.N., McCown, T.J. and Kafri, T.: « Transactivator and structurally optimized inducible lentiviral vectors » Mol Ther 10 (2004) 585-596.
Israeli, D., Benchaouir, R., Ziaei, S., Rameau, P., Gruszczynski, C., Peltekian, E., Danos, O. and Garcia, L.: « FGF6 mediated expansion of a resident subset of cells with SP phenotype in the C2C12 myogenic line » J Cell Physiol 201 (2004) 409-419.
Kichler, A.: « Gene transfer with modified polyethylenimines » J Gene Med 6 (2004) S3-10.
Lefevre, C., Bouadjar, B., Karaduman, A., Jobard, F., Saker, S., Ozguc, M., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Mutations in ichthyin a new gene on chromosome 5q33 in a new form of autosomal recessive congenital ichthyosis » Hum Mol Genet 18 (2004) 18.
Lemoine, F.M., Mesel-Lemoine, M., Cherai, M., Gallot, G., Vie, H., Leclercq, V., Trebeden-Negre, H., Mammes, O., Boyer, O., Noguiez-Hellin, P. and Klatzmann, D.: « Efficient transduction and selection of human T-lymphocytes with bicistronic Thy1/HSV1-TK retroviral vector produced by a human packaging cell line » J Gene Med 6 (2004) 374-86.
Martineau, Y., Le Bec, C., Monbrun, L., Allo, V., Chiu, I.M., Danos, O., Moine, H., Prats, H. and Prats, A.C.: « Internal ribosome entry site structural motifs conserved among mammalian Fibroblast growth factor 1 alternatively spliced mRNAs » Mol Cell Biol 24 (2004) 7622-7635.
Merten, O.-W.: « State-of-art of the production of retroviral vectors » J Gene Med 6 (2004) S105-24.
Millot, G.A., Vainchenker, W., Dumenil, D. and Svinarchuk, F.: « Differential signalling of NH2-terminal flag-labelled thrombopoietin receptor activated by TPO or anti-FLAG antibodies » Cell Signal 16 (2004) 355-63.
Movassagh, M., Spatz, A., Davoust, J., Lebecque, S., Romero, P., Pittet, M., Rimoldi, D., Lienard, D., Gugerli, O., Ferradini, L., Robert, C., Avril, M.F., Zitvogel, L. and Angevin, E.: « Selective accumulation of mature DC-Lamp+ dendritic cells in tumor sites is associated with efficient T-cell-mediated antitumor response and control of metastatic dissemination in melanoma » Cancer Res 64 (2004) 2192-8.
Perez, N., Bigey, P., Scherman, D., Danos, O., Piechaczyk, M. and Pelegrin, M.: « Regulatable systemic production of monoclonal antibodies by in vivo muscle electroporation » Genet Vaccines Ther 2 (2004) 2.
Raslova, H., Komura, E., Le Couédic, J.P., Larbret, F., Debili, N., Feunteun, J., Danos, O., Albagli, O., Vainchenker, W. and Favier, R.: « FLI1 monoallelic expression combined with its hemizygous loss underlies Paris-Trousseau/Jacobsen thrombopenia. » J Clin Invest 114 (2004) 77-84.
Salaun, B., de Saint-Vis, B., Pacheco, N., Pacheco, Y., Riesler, A., Isaac, S., Leroux, C., Clair-Moninot, V., Pin, J.J., Griffith, J., Treilleux, I., Goddard, S., Davoust, J., Kleijmeer, M. and Lebecque, S.: « CD208/dendritic cell-lysosomal associated membrane protein is a marker of normal and transformed type II pneumocytes » Am J Pathol 164 (2004) 861-71.
Taveau, M., Stockholm, D., Marchand, S., Roudaut, C., Le Bert, M. and Richard, I.: « Bidirectional transcriptional activity of the Pgk1 promoter and transmission ratio distortion in Capn3-deficient mice » Genomics 84 (2004) 592-5.
Tazir, M., Azzedine, H., Assami, S., Sindou, P., Nouioua, S., Zemmouri, R., Hamadouche, T., Chaouch, M., Feingold, J., Vallat, J.M., Leguern, E. and Grid, D.: « Phenotypic variability in autosomal recessive axonal Charcot-Marie-Tooth disease due to the R298C mutation in lamin A/C » Brain 127 (2004) 154-63.
Topcu, M., Jobard, F., Halliez, S., Coskun, T., Yalcinkayal, C., Gerceker, F.O., Wanders, R.J., Prud'homme, J.F., Lathrop, M., Ozguc, M. and Fischer, J.: « L-2-Hydroxyglutaric aciduria: identification of a mutant gene C14orf160, localized on chromosome 14q22.1 » Hum Mol Genet 13 (2004) 2803-11.
Vallat, J.M., Grid, D., Magdelaine, C., Sturtz, F. and Tazir, M.: « Autosomal recessive forms of Charcot-Marie-Tooth disease » Curr Neurol Neurosci Rep 4 (2004) 413-9.

2003

Baldeschi, C., Gache, Y., Rattenholl, A., Bouille, P., Danos, O., Ortonne, J.P., Bruckner-Tuderman, L. and Meneguzzi, G.: « Genetic correction of canine dystrophic epidermolysis bullosa mediated by retroviral vectors » Hum Mol Genet 12 (2003) 1897-905.
Brissault, B., Kichler, A., Guis, C., Leborgne, C., Danos, O. and Cheradame, H.: « Synthesis of linear polyethylenimine derivatives for DNA transfection » Bioconjug Chem 14 (2003) 581-7.
Chalouni, C., Banchereau, J., Vogt, A.B., Pascual, V. and Davoust, J.: « Human germinal center B cells differ from naive and memory B cells by their aggregated MHC class II-rich compartments lacking HLA-DO » Int Immunol 15 (2003) 457-66.
Chantret, I., Dancourt, J., Dupre, T., Delenda, C., Bucher, S., Vuillaumier-Barrot, S., Ogier de Baulny, H., Peletan, C., Danos, O., Seta, N., Durand, G., Oriol, R., Codogno, P. and Moore, S.E.: « A deficiency in dolichyl-P-glucose:Glc1Man9GlcNAc2-PP-dolichyl alpha3-glucosyltransferase defines a new subtype of congenital disorders of glycosylation » J Biol Chem 278 (2003) 9962-71.
Chaouch, M., Allal, Y., De Sandre-Giovannoli, A., Vallat, J.M., Amer-el-Khedoud, A., Kassouri, N., Chaouch, A., Sindou, P., Hammadouche, T., Tazir, M., Levy, N. and Grid, D.: « The phenotypic manifestations of autosomal recessive axonal Charcot-Marie-Tooth due to a mutation in Lamin A/C gene » Neuromuscul Disord 13 (2003) 60-7.
Combaret, L., Bechet, D., Claustre, A., Taillandier, D., Richard, I. and Attaix, D.: « Down-regulation of genes in the lysosomal and ubiquitin-proteasome proteolytic pathways in calpain-3-deficient muscle » Int J Biochem Cell Biol 35 (2003) 676-84.
De Sandre-Giovannoli, A., Chaouch, M., Boccaccio, I., Bernard, R., Delague, V., Grid, D., Vallat, J.M., Levy, N. and Megarbane, A.: « Phenotypic and genetic exploration of severe demyelinating and secondary axonal neuropathies resulting from GDAP1 nonsense and splicing mutations » J Med Genet 40 (2003) e87.
Douar, A.M., Poulard, K. and Danos, O.: « Deleterious effect of peptide insertions in a permissive site of the AAV2 capsid » Virology 309 (2003) 203-8.
Fougerousse, F., Gonin, P., Durand, M., Richard, I. and Raymackers, J.M.: « Force impairment in calpain 3-deficient mice is not correlated with mechanical disruption » Muscle Nerve 27 (2003) 616-623.
Fruci, D., Lauvau, G., Saveanu, L., Amicosante, M., Butler, R.H., Polack, A., Ginhoux, F., Lemonnier, F., Firat, H. and van Endert, P.M.: « Quantifying recruitment of cytosolic peptides for HLA class I presentation: impact of TAP transport » J Immunol 170 (2003) 2977-84.
Ginhoux, F., Doucet, C., Leboeuf, M., Lemonnier, F.A., Danos, O., Davoust, J. and Firat, H.: « Identification of an HLA-A*0201-restricted epitopic peptide from human dystrophin: application in duchenne muscular dystrophy gene therapy » Mol Ther 8 (2003) 274-83.
Gross, D.A., Leboeuf, M., Gjata, B., Danos, O. and Davoust, J.: « CD4+CD25+ regulatory T cells inhibit immune mediated transgene rejection » Blood 102 (2003) 4326-4328.
Guermonprez, P., Saveanu, L., Kleijmeer, M., Davoust, J., Van Endert, P. and Amigorena, S.: « ER-phagosome fusion defines an MHC class I cross-presentation compartment in dendritic cells » Nature 425 (2003) 397-402.
Jobard, F., Bouadjar, B., Caux, F., Hadj-Rabia, S., Has, C., Matsuda, F., Weissenbach, J., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Identification of mutations in a new gene encoding a FERM family protein with a pleckstrin homology domain in Kindler syndrome » Hum Mol Genet 12 (2003) 925-35.
Karsenty, E., Barillot, E., Tosser-Klopp, G., Lahbib-Mansais, Y., Milan, D., Hatey, F., Cirera, S., Sawera, M., Jorgensen, C.B., Chowdhary, B., Fredholm, M., Wimmers, K., Ponsuksili, S., Davoli, R., Fontanesi, L., Braglia, S., Zambonelli, P., Bigi, D., Neuenschwander, S. and Gellin, J.L.: « The GENETPIG database: a tool for comparative mapping in pig (Sus scrofa) » Nucleic Acids Research 31 (2003) 138-141.
Kichler, A., Bechinger, B. and Danos, O.: « Des peptides cationiques antibactériens comme vecteurs de transfert de gènes » Med Sci (Paris) 19 (2003) 1046-47.
Kichler, A., Leborgne, C., März, J., Danos, O. and Bechinger, B.: « Histidine-rich amphipathic peptide antibiotics promote efficient delivery of DNA into mammalian cells » Proc Natl Acad Sci USA 100 (2003) 1564-1568.
Kichler, A., Sabourault, N., Decor, R., Leborgne, C., Schmutz, M., Valleix, A., Danos, O., Wagner, A. and Mioskowski, C.: « Preparation and evaluation of a new class of gene transfer reagents: poly(-alkylaminosiloxanes) » J Control Release 93 (2003) 403-14.
Laderach, D., Compagno, D., Danos, O., Vainchenker, W. and Galy, A.: « RNA Interference Shows Critical Requirement for NF-kappaB p50 in the Production of IL-12 by Human Dendritic Cells » J Immunol 171 (2003) 1750-7.
Lefèvre, C., Audebert, S., Jobard, F., Bouadjar, B., Lakhdar, H., Boughdene-Stambouli, O., Blanchet-Bardon, C., Heilig, R., Foglio, M., Weissenbach, J., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Mutations in the transporter ABCA12 are associated with lamellar ichthyosis type 2 » Hum Mol Genet 12 (2003) 2369-78.
Marchand, S., Hajdari, P., Hackman, P., Udd, B. and Richard, I.: « Touch-down method for high-performance sequencing of polymerase chain reaction products » Anal Biochem 315 (2003) 270-2.
Marrakchi, S., Audebert, S., Bouadjar, B., Has, C., Lefevre, C., Munro, C., Cure, S., Jobard, F., Morlot, S., Hohl, D., Prud'homme, J.F., Zahaf, A., Turki, H. and Fischer, J.: « Novel mutations in the gene encoding secreted lymphocyte antigen-6/urokinase-type plasminogen activator receptor-related protein-1 (SLURP-1) and description of five ancestral haplotypes in patients with Mal de Meleda » J Invest Dermatol 120 (2003) 351-5.
Merten, O.-W.: « Métabolisme cellulaire et besoin nutritif des cellules en culture » In: Barlovatz-Meinon, G. and Adolphe, M. (Eds.), Techniques en Cultures de cellules animales: méthodologies, applications. Les Editions Inserm, Paris, 2003, pp. 99-160.
Nabbout, R., Gennaro, E., Dalla Bernardina, B., Dulac, O., Madia, F., Bertini, E., Capovilla, G., Chiron, C., Cristofori, G., Elia, M., Fontana, E., Gaggero, R., Granata, T., Guerrini, R., Loi, M., La Selva, L., Lispi, M.L., Matricardi, A., Romeo, A., Tzolas, V., Valseriati, D., Veggiotti, P., Vigevano, F., Vallee, L., Dagna Bricarelli, F., Bianchi, A. and Zara, F.: « Spectrum of SCN1A mutations in severe myoclonic epilepsy of infancy » Neurology 60 (2003) 1961-7.
Nabbout, R., Kozlovski, A., Gennaro, E., Bahi-Buisson, N., Zara, F., Chiron, C., Bianchi, A., Brice, A., Leguern, E. and Dulac, O.: « Absence of mutations in major GEFS+ genes in myoclonic astatic epilepsy » Epilepsy Res 56 (2003) 127-33.
Pagès, J.C. and Danos, O.: « Retrovectors go forward » In: Rolland, A. and Sullivan, S.M. (Eds.), Pharmaceutical Gene Delivery Systems. Marcel Dekker, Inc, Basel, 2003, pp. 215-244.
Rohrlich, P.S., Cardinaud, S., Firat, H., Lamari, M., Briand, P., Escriou, N. and Lemonnier, F.A.: « HLA-B*0702 transgenic, H-2K(b)D(b) double-knockout mice: phenotypical and functional characterization in response to influenza virus » Int Immunol 15 (2003) 765-772.
Sander, T., Windemuth, C., Schulz, H., Saar, K., Gennaro, E., Riggio, C., Bianchi, A., Zara, F., Rudolf, G., Picard, F., Bulteau, C., Kaminska, A., Cieuta, C., Prud'homme, J.F., Dulac, O., Bate, L., Robinson, R., Gardiner, R.M., Covanis, A., De Haan, G.J., Janssen, G.A., Van Erp, M.G., Boezeman, E.H., Lindhout, D., Heils, A., Nurnberg, P. and Janz, D.: « Exploration of a putative susceptibility locus for idiopathic generalized epilepsy on chromosome 8p12 » Epilepsia 44 (2003) 32-9.
Sandre-Giovannoli, A.D., Chaouch, M., Boccaccio, I., Bernard, R., Delague, V., Grid, D., Vallat, J.M., Levy, N. and Megarbane, A.: « Phenotypic and genetic exploration of severe demyelinating and secondary axonal neuropathies resulting from GDAP1 nonsense and splicing mutations » Journal Of Medical Genetics 40 (2003).
Scardino, A., Correale, P., Firat, H., Pellegrini, M., Kosmatopoulos, K., Opolon, P., Alves, P., Zurbriggen, R., Gluck, R., Lemonnier, F.A., Francini, G. and Cusi, M.G.: « In vivo study of the GC90/IRIV vaccine for immune response and autoimmunity into a novel humanised transgenic mouse » Br J Cancer 89 (2003) 199-205.
Seifert, U., Maranon, C., Shmueli, A., Desoutter, J.F., Wesoloski, L., Janek, K., Henklein, P., Diescher, S., Andrieu, M., de la Salle, H., Weinschenk, T., Schild, H., Laderach, D., Galy, A., Haas, G., Kloetzel, P.M., Reiss, Y. and Hosmalin, A.: « An essential role for tripeptidyl peptidase in the generation of an MHC class I epitope » Nat Immunol 4 (2003) 375-9.
Stumptner-Cuvelette, P., Jouve, M., Helft, J., Dugast, M., Glouzman, A.S., Jooss, K., Raposo, G. and Benaroch, P.: « Human immunodeficiency virus-1 Nef expression induces intracellular accumulation of multivesicular bodies and major histocompatibility complex class II complexes: potential role of phosphatidylinositol 3-kinase » Mol Biol Cell 14 (2003) 4857-70.
Taveau, M., Bourg, N., Sillon, G., Roudaut, C., Bartoli, M. and Richard, I.: « Calpain 3 is activated through autolysis within the active site and lyses sarcomeric and sarcolemmal components » Mol Cell Biol 23 (2003) 9127-35.
Van Den Bergh, P.Y., Bouquiaux, O., Verellen, C., Marchand, S., Richard, I., Hackman, P. and Udd, B.: « Tibial muscular dystrophy in a Belgian family » Ann Neurol 54 (2003) 248-51.

2002

Bessis, N., Doucet, C., Cottard, V., Douar, A.M., Firat, H., Jorgensen, C., Mezzina, M. and Boissier, M.C.: « Gene therapy for rheumatoid arthritis » J Gene Med 4 (2002) 581-91.
Chantret, I., Dupre, T., Delenda, C., Bucher, S., Dancourt, J., Barnier, A., Charollais, A., Heron, D., Bader-Meunier, B., Danos, O., Seta, N., Durand, G., Oriol, R., Codogno, P. and Moore, S.E.: « Congenital disorders of glycosylation type Ig is defined by a deficiency in dolichyl-P-mannose:Man7GlcNAc2-PP-dolichyl mannosyltransferase » J Biol Chem 277 (2002) 25815-25822.
De Lonlay, P., Mugnier, C., Sanlaville, D., Chantrel-Groussard, K., Benit, P., Lebon, S., Chretien, D., Kadhom, N., Saker, S., Gyapay, G., Romana, S., Weissenbach, J., Munnich, A., Rustin, P. and Rotig, A.: « Cell complementation using Genebridge 4 human:rodent hybrids for physical mapping of novel mitochondrial respiratory chain deficiency genes » Hum Mol Genet 11 (2002) 3273-81.
De Sandre-Giovannoli, A., Chaouch, M., Kozlov, S., Vallat, J.M., Tazir, M., Kassouri, N., Szepetowski, P., Hammadouche, T., Vandenberghe, A., Stewart, C.L., Grid, D. and Levy, N.: « Homozygous defects in LMNA, encoding lamin A/C nuclear-envelope proteins, cause autosomal recessive axonal neuropathy in human (Charcot-Marie-Tooth disorder type 2) and mouse » Am J Hum Genet 70 (2002) 726-736 (Erratum in: Am J Hum Genet 2002 Apr;70(4):1075.
Delenda, C., Audit, M. and Danos, O.: « Biosafety issues in lentivector production » In: Trono, D. (Ed.), Lentiviral Vectors. Springer-Verlag, Berlin, 2002, pp. 123-141.
Duan, M.L., Bordet, T., Mezzina, M., Kahn, A. and Ulfendahl, M.: « Adenoviral and adeno-associated viral vector mediated gene transfer in the guinea pig cochlea » Neuroreport 13 (2002) 1295-1299.
Feasson, L., Stockholm, D., Freyssenet, D., Richard, I., Duguez, S., Beckmann, J.S. and Denis, C.: « Molecular adaptations of neuromuscular disease-associated proteins in response to eccentric exercise in human skeletal muscle » J Physiol 543 (2002) 297-306.
Firat, H., Cochet, M., Rohrlich, P.S., Garcia-Pons, F., Darche, S., Danos, O., Lemonnier, F.A. and Langlade-Demoyen, P.: « Comparative analysis of the CD8(+) T cell repertoires of H-2 class I wild-type/HLA-A2.1 and H-2 class I knockout/HLA-A2.1 transgenic mice » Int Immunol 14 (2002) 925-934.
Firat, H., Zennou, V., Garcia-Pons, F., Ginhoux, F., Cochet, M., Danos, O., Lemonnier, A.F., Langlade-Demoyen, P. and Charneau, P.: « Use of a lentiviral flap vector for induction of CTL immunity against melanoma. Perspectives for immunotherapy » J Gene Med 4 (2002) 38-45.
Fougerousse, F., Durand, M., Lopez, S., Suel, L., Demignon, J., Thornton, C., Ozaki, H., Kawakami, K., Barbet, P., Beckmann, J.S. and Maire, P.: « Six and Eya expression during human somitogenesis and MyoD gene family activation » J Muscle Res Cell Motil 23 (2002) 255-264.
Genin, E., Huebner, A., Jaillard, C., Faure, A., Halaby, G., Saka, N., Clark, A.J.L., Durand, P., Begeot, M. and Naville, D.: « Linkage of one gene for familial glucocorticoid deficiency type 2 (FGD2) to chromosome 8q and further evidence of heterogeneity » Human Genetics 111 (2002) 428-434.
Hackman, P., Vihola, A., Haravuori, H., Marchand, S., Sarparanta, J., De Seze, J., Labeit, S., Witt, C., Peltonen, L., Richard, I. and Udd, B.: « Tibial muscular dystrophy is a titinopathy caused by mutations in TTN, the gene encoding the giant skeletal-muscle protein titin » Am J Hum Genet 71 (2002) 492-500.
Hamel, Y., Blake, N., Gabrielsson, S., Haigh, T., Jooss, K., Martinache, C., Caillat-Zucman, S., Rickinson, A.B., Hacein-Bey, S., Fischer, A. and Cavazzana-Calvo, M.: « Adenovirally transduced dendritic cells induce bispecific cytotoxic T lymphocyte responses against adenovirus and cytomegalovirus pp65 or against adenovirus and Epstein-Barr virus EBNA3C protein: a novel approach for immunotherapy » Hum Gene Ther 13 (2002) 855-866.
Hernandez, J., Garcia-Pons, F., Lone, Y.C., Firat, H., Schmidt, J.D., Langlade-Demoyen, P. and Zanetti, M.: « Identification of a human telomerase reverse transcriptase peptide of low affinity for HLA A2.1 that induces cytotoxic T lymphocytes and mediates lysis of tumor cells » Proc Natl Acad Sci USA 99 (2002) 12275-12280.
Jobard, F., Lefèvre, C., Karaduman, A., Blanchet-Bardon, C., Emre, S., Weissenbach, J., Özgüc, M., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Lipoxygenase-3 (ALOXE3) and 12(R)-lipoxygenase (ALOX12B) are mutated in non-bullous congenital ichthyosiform erythroderma (NCIE) linked to chromosome 17p13.1 » Hum Mol Genet 11 (2002) 107-113.
Kichler, A., Chillon, M., Leborgne, C., Danos, O. and Frisch, B.: « Intranasal gene delivery with a polyethylenimine-PEG conjugate » J Control Release 81 (2002) 379-388.
Kissa, K., Mordelet, E., Soudais, C., Kremer, E., Demeneix, B., Brulet, P. and Coen, L.: « In vivo neuronal tracing with GFP-TTC gene delivery » Mol Cell Neurosci 20 (2002) 627-637.
Lambolez, F., Jooss, K., Vasseur, F. and Sarukhan, A.: « Tolerance induction to self antigens by peripheral dendritic cells » Eur J Immunol 32 (2002) 2588-2597.
Li Duan, M., Bordet, T., Mezzina, M., Kahn, A. and Ulfendahl, M.: « Adenoviral and adeno-associated viral vector mediated gene transfer in the guinea pig cochlea » Neuroreport 13 (2002) 1295-1299.
Mahe, E., Lahfa, M., Mansouri, S., Mosharraf-Olmolk, H., Le Rebours, J., Prud'homme, J.F. and Fischer, J.: « Physicians' response to a letter to confirm diagnosis in a genetic study of psoriasis » Eur J Dermatol 12 (2002) 66-69.
Martin-Touaux, E., Puech, J.P., Chateau, D., Emiliani, C., Kremer, E.J., Raben, N., Tancini, B., Orlacchio, A., Kahn, A. and Poenaru, L.: « Muscle as a putative producer of acid alpha-glucosidase for glycogenosis type II gene therapy » Hum Mol Genet 11 (2002) 1637-1645.
Mathews, L.C., Gray, J.T., Gallagher, M.R. and Snyder, R.O.: « Recombinant adeno-associated viral vector production using stable packaging and producer cell lines » In: Phillips, I. (Ed.), Gene Therapy Methods. Academic Press, San Diego, 2002, pp. 393-413.
Merten, O.-W.: « Development of serum-free media for cell growth and production of viruses / viral vaccines – safety issues of animal products used in serum-free media » In: Brown, F., Hendriksen, C., Sesardic, D. and Cussler, K. (Eds.), Advancing Science and Elimination of the Use of Laboratory Animals for Development and Control of Vaccines and Hormones – Symposium, Utrecht, November 2001: Proceedings. S. Karger AG, Basel, 2002, pp. 233-257.
Mezzina, M. and Danos, O.: « Five years of vector service for gene therapy » Trends Genet 18 (2002) 118-119.
Morante-Redolat, J.M., Gorostidi-Pagola, A., Piquer-Sirerol, S., Saenz, A., Poza, J.J., Galan, J., Gesk, S., Sarafidou, T., Mautner, V.F., Binelli, S., Staub, E., Hinzmann, B., French, L., Prud'homme, J.F., Passarelli, D., Scannapieco, P., Tassinari, C.A., Avanzini, G., Marti-Masso, J.F., Kluwe, L., Deloukas, P., Moschonas, N.K., Michelucci, R., Siebert, R., Nobile, C., Perez-Tur, J. and Lopez de Munain, A.: « Mutations in the LGI1/Epitempin gene on 10q24 cause autosomal dominant lateral temporal epilepsy » Hum Mol Genet 11 (2002) 1119-1128.
Moulard, B., Genton, P., Grid, D., Jeanpierre, M., Ouazzani, R., Mrabet, A., Morris, M., LeGuern, E., Dravet, C., Mauguiere, F., Utermann, B., Baldy-Moulinier, M., Belaidi, H., Bertran, F., Biraben, A., Ali Cherif, A., Chkili, T., Crespel, A., Darcel, F., Dulac, O., Geny, C., Humbert-Claude, V., Kassiotis, P., Buresi, C. and Malafosse, A.: « Haplotype study of West European and North African Unverricht-Lundborg chromosomes: evidence for a few founder mutations » Hum Genet 111 (2002) 255-62.
Nabbout, R., Prud'homme, J.F., Herman, A., Feingold, J., Brice, A., Dulac, O. and LeGuern, E.: « A locus for simple pure febrile seizures maps to chromosome 6q22-q24 » Brain 125 (2002) 2668-80.
Perez, N., Plence, P., Millet, V., Greuet, D., Minot, C., Noel, D., Danos, O., Jorgensen, C. and Apparailly, F.: « Tetracycline transcriptional silencer tightly controls transgene expression after in vivo intramuscular electrotransfer: application to interleukin 10 therapy in experimental arthritis » Hum Gene Ther 13 (2002) 2161-72.
Robertson, A., Perea, J., Tolmachova, T., Thomas, P.K. and Huxley, C.: « Effects of mouse strain, position of integration and tetracycline analogue on the tetracycline conditional system in transgenic mice » Gene 282 (2002) 65-74.
Sander, T., Windemuth, C., Schulz, H., Saar, K., Gennaro, E., Bianchi, A., Zara, F., Bulteau, C., Kaminska, A., Ville, D., Cieuta, C., Prud'homme, J.F., Dulac, O., Bate, L., Gardiner, R.M., de Haan, G.J., Janssen, G.A., Witte, J., Halley, D.J., Lindhout, D., Wienker, T.F. and Janz, D.: « No evidence for a susceptibility locus for idiopathic generalized epilepsy on chromosome 18q21.1 » Am J Med Genet 114 (2002) 673-8.
Schneider, H., Muhle, C., Douar, A.M., Waddington, S., Jiang, Q.J., Von der Mark, K., Coutelle, C. and Rascher, W.: « Sustained delivery of therapeutic concentrations of human clotting factor IX – a comparison of adenoviral and AAV vectors administered in utero » J Gene Med 4 (2002) 46-53.
Spencer, M.J., Guyon, J.R., Sorimachi, H., Potts, A., Richard, I., Herasse, M., Chamberlain, J., Dalkilic, I., Kunkel, L.M. and Beckmann, J.S.: « Stable expression of calpain 3 from a muscle transgene in vivo: immature muscle in transgenic mice suggests a role for calpain 3 in muscle maturation » Proc Natl Acad Sci USA 99 (2002) 8874-8879.
Taveau, M., Stockholm, D., Spencer, M. and Richard, I.: « Quantification of splice variants using molecular beacon or scorpion primers » Anal Biochem 305 (2002) 227-235.
Tazir, M., Vallat, J.M., Bomont, P., Zemmouri, R., Sindou, P., Assami, S., Nouioua, S., Hammadouche, T., Grid, D. and Koenig, M.: « Genetic heterogeneity in giant axonal neuropathy: an Algerian family not linked to chromosome 16q24.1 » Neuromuscul Disord 12 (2002) 849-52.
Viollet, L., Barois, A., Rebeiz, J.G., Rifai, Z., Burlet, P., Zarhrate, M., Vial, E., Dessainte, M., Estournet, B., Kleinknecht, B., Pearn, J., Adams, R.D., Urtizberea, J.A., Cros, D.P., Bushby, K., Munnich, A. and Lefebvre, S.: « Mapping of autosomal recessive chronic distal spinal muscular atrophy to chromosome 11q13 » Ann Neurol 51 (2002) 585-592.
Windemuth, C., Schulz, H., Saar, K., Gennaro, E., Bianchi, A., Zara, F., Bulteau, C., Kaminska, A., Ville, D., Cieuta, C., Nabbout-Tarantino, R., Prud'homme, J.F., Dulac, O., Bate, L., Gardiner, R., Lindhout, D., Wienker, T., Janz, D. and Sander, T.: « No evidence for a susceptibility locus for idiopathic generalized epilepsy on chromosome 5 in families with typical absence seizures » Epilepsy Res 51 (2002) 23-9.
Yates, F., Malassis-Seris, M., Stockholm, D., Bouneaud, C., Larousserie, F., Noguiez-Hellin, P., Danos, O., Kohn, D.B., Fischer, A., De Villartay, J.P. and Cavazzana-Calvo, M.: « Gene therapy of RAG-2-/-mice: sustained correction of the immunodeficiency » Blood 100 (2002) 3942-3949.

2001

Achard, F., Vaysseix, G. and Barillot, E.: « XML, bioinformatics and data integration » Bioinformatics 17 (2001) 115-125.
Aoki, M., Liu, J., Richard, I., Bashir, R., Britton, S., Keers, S.M., Oeltjen, J., Brown, H.E., Marchand, S., Bourg, N., Beley, C., McKenna-Yasek, D., Arahata, K., Bohlega, S., Cupler, E., Illa, I., Majneh, I., Barohn, R.J., Urtizberea, J.A., Fardeau, M., Amato, A., Angelini, C., Bushby, K., Beckmann, J.S. and Brown, R.H., Jr.: « Genomic organization of the dysferlin gene and novel mutations in Miyoshi myopathy » Neurology 57 (2001) 271-8.
Baghdiguian, S., Richard, I., Martin, M., Coopman, P., Beckmann, J.S., Mangeat, P. and Lefranc, G.: « Pathophysiology of limb girdle muscular dystrophy type 2A: hypothesis and new insights into the IkappaBalpha/NF-kappaB survival pathway in skeletal muscle » J Mol Med 79 (2001) 254-61.
Balsa, A., Barrera, P., Westhovens, R., Alves, H., Maenaut, K., Pascual-Salcedo, D., Cornelis, F., Bardin, T., Riente, L., Radstake, T.R.D.J., De Almeida, G., Lepage, V., Stravopoulos, C., Spaepen, M., Lopes-Vaz, A., Charron, D., Martinez, M., Prud'homme, J.F., Migliorini, P. and Fritz, P.: « Clinical and immunogenetic characteristics of European multicase rheumatoid arthritis families » Ann Rheum Dis 60 (2001) 573-576.
Barrera, P., Balsa, A., Alves, H., Westhovens, R., Maenaut, K., Cornelis, F., Fritz, P., Bardin, T., Ceu Maia, M., Lopes-Vaz, A., Pascual Salcedo, D., de la Concha, E., Radstake, T., van de Putte, L.B., Migliorini, P., Prud'homme, J.F., Charron, D., Spyropoulou, M., Mendes, A., Spaepen, M., Martinez, M. and Stavropoulos, C.: « Noninherited maternal antigens do not increase the susceptibility for familial rheumatoid arthritis. European Consortium on Rheumatoid Arthritis Families (ECRAF) » J Rheumatol 28 (2001) 968-974.
Barrera, P., Faure, S., Prud'homme, J.F., Balsa, A., Migliorini, P., Chimenti, D., Radstake, T.R., van de Putte, L.B., Pascual-Salcedo, D., Westhovens, R., Maenaut, K., Alves, H., Lopes-Vaz, A., Stravopoulos, C., Spyropoulou, M., Fritz, P., Bardin, T., Charron, D., Lepage, V., Alibert, Martinez, M. and Cornelis, F.: « European genetic study on rheumatoid arthritis: is there a linkage of the interleukin-1 (IL-1), IL-10 or IL-4 genes to RA? » Clin Exp Rheumatol 19 (2001) 709-714.
Baulac, S., Huberfeld, G., Gourfinkel-An, I., Mitropoulou, G., Beranger, A., Prud'homme, J.F., Baulac, M., Brice, A., Bruzzone, R. and LeGuern, E.: « First genetic evidence of GABAa receptor dysfunction in epilepsy: A mutation in the gamma2-subunit gene » Nat Genet 28 (2001) 46-48.
Baulac, S., Picard, F., Herman, A., Feingold, J., Genin, E., Hirsch, E., Prud'homme, J.F., Baulac, M., Brice, A. and LeGuern, E.: « Evidence for a digenic inheritance in a family with both febrile convulsions and temporal lobe epilepsy implicating chromosomes 18qter and 1q25-q31 » Ann Neurol 49 (2001) 786-792.
Ben-Arieh, S.V., Zimerman, B., Smorodinsky, N.I., Yaacubovicz, M., Schechter, C., Bacik, I., Gibbs, J., Bennink, J.R., Yewdell, J.W., Coligan, J.E., Firat, H., Lemonnier, F. and Ehrlich, R.: « Human cytomegalovirus protein US2 interferes with the expression of human HFE, a nonclassical class I major histocompatibility complex molecule that regulates iron homeostasis » J Virol 75 (2001) 10557-62.
Brockington, M., Yuva, Y., Prandini, P., Brown, S.C., Torelli, S., Benson, M.A., Herrmann, R., L.V., A., Bashir, R., Burgunder, J.M., Fallet, S., Romero, N., Fardeau, M., Straub, V., Storey, G., Pollitt, C., Richard, I., Sewry, C.A., Bushby, K., Voit, T., Blake, D.J. and Muntoni, F.: « Mutations in the fukutin-related protein gene (FKRP) identify limb girdle muscular dystrophy 2I as a milder allelic variant of congenital muscular dystrophy MDC1C » Hum Mol Genet 10 (2001) 2851-2859.
Chillon, M. and Kremer, E.J.: « Trafficking and propagation of canine adenovirus vectors lacking a known integrin-interacting motif » Human Gene Therapy 12 (2001) 1815-1823.
Coutelle, C., Themis, M., Schneider, H., Kieserud, T., Cook, T., Douar, A.M., Hanson, M., Pavirani, A. and Rodeck, C.: « Fetal somatic gene therapy – A preventive approach to the treatment of genetic disease » In: Holzgreve, W. and Lessl, M. (Eds.), Stem cells from cord blood, in utero stem cell development, and transplantation-inclusive gene therapy. Springer-Verlag, Berlin, 2001, pp. 99-114.
Douar, A.M. and Danos, O.: « Transfert de gène au moyen de vecteurs adéno-associés » In: Cohen-Haguenauer, O. (Ed.), Thérapie Génique. Editions Tec &amp; Doc/Editions Médicales Internationales, Paris, 2001, pp. 145-154.
Drittanti, L., Jenny, C., Poulard, K., Samba, A., Manceau, P., Soria, N., Vincent, N., Danos, O. and Vega, M.: « Optimised helper virus-free production of high-quality adeno-associated virus vectors » J Gene Med 3 (2001) 59-71.
Firat, H., Tourdot, S., Ureta-Vidal, A., Scardino, A., Suhrbier, A., Buseyne, F., Riviere, Y., Danos, O., Michel, M.L., Kosmatopoulos, K. and Lemonnier, F.A.: « Design of a polyepitope construct for the induction of HLA-A0201-restricted HIV 1-specific CTL responses using HLA-A*0201 transgenic, H-2 class I KO mice » Eur J Immunol 31 (2001) 3064-3074.
Fischer, J., Bouadjar, B., Heilig, R., Huber, M., Lefevre, C., Jobard, F., Macari, F., Bakija-Konsuo, A., Ait-Belkacem, F., Weissenbach, J., Lathrop, M., Hohl, D. and Prud'homme, J.F.: « Mutations in the gene encoding SLURP-1 in Mal de Meleda » Hum Mol Genet 10 (2001) 875-880.
Fougerousse, F., Edom-Vovard, F., Merkulova, T., Ott, M.O., Durand, M., Butler-Browne, G. and Keller, A.: « The muscle-specific enolase is an early marker of human myogenesis » J Muscle Res Cell Motil 22 (2001) 535-544.
Gücüyener, K., Özgül, K., Paternotte, C., Erdem, H., Prud'homme, J.F., Özgüc, M. and Topaloglu, H.: « Autosomal recessive spastic ataxia of Charlevoix-Saguenay in two unrelated Turkish families » Neuropediatrics 32 (2001) 142-146.
Haravuori, H., Vihola, A., Straub, V., Auranen, M., Richard, I., Marchand, S., Voit, T., Labeit, S., Somer, H., Peltonen, L., Beckmann, J.S. and Udd, B.: « Secondary calpain 3 deficiency in 2q-linked muscular dystrophy: Titin is the candidate gene » Neurology 56 (2001) 869-877.
Jooss, K., Gjata, B., Danos, O., Von Boehmer, H. and Sarukhan, A.: « Regulatory function of in vivo anergized CD4+ T cells » Proc Natl Acad Sci USA 98 (2001) 8738-8743.
Kichler, A., Leborgne, C., Coeytaux, E. and Danos, O.: « Polyethylenimine-mediated gene delivery: a mechanistic study » J Gene Med 3 (2001) 135-144.
Lefèvre, C., Blanchet-Bardon, C., Jobard, F., Bouadjar, B., Stalder, J.F., Cure, S., Hoffmann, A., Prud'homme, J.F. and Fischer, J.: « Novel point mutations, deletions, and polymorphisms in the cathepsin C gene in nine families from Europe and North Africa with Papillon-Lefèvre syndrome » J Invest Dermatol 117 (2001) 1657-1661.
Lefèvre, C., Jobard, F., Caux, F., Bouadjar, B., Karaduman, A., Heilig, R., Lakhdar, H., Wollenberg, A., Verret, J.L., Weissenbach, J., Özgüc, M., Lathrop, M., Prud'homme, J.F. and Fischer, J.: « Mutations in CGI-58, the gene encoding a new protein of the esterase/Lipase/Thioesterase subfamily, in Chanarin-Dorfman syndrome » Am J Hum Genet 69 (2001) 1002-1012.
Martinez-Arca, S., Coco, S., Mainguy, G., Schenk, U., Alberts, P., Bouille, P., Mezzina, M., Prochiantz, A., Matteoli, M., Louvard, D. and Galli, T.: « A common exocytotic mechanism mediates axonal and dendritic outgrowth » J Neurosci 21 (2001) 3830-3838.
Merten, O.-W., Cruz, P.E., Rochette, C., Geny-Fiamma, C., Bouquet, C., Gonçalves, D., Danos, O. and Carrondo, M.J.T.: « Comparison of different bioreactor systems for the production of high titer retroviral vectors » Biotechnol Prog 17 (2001) 326-335.
Merten, O.-W., Landric, L. and Danos, O.: « Influence of the metabolic state of packaging cells on retroviral vector production » In: Merten, O.W., Mattanovich, D., Lang, C., Larsson, G., Neubauer, P., Porro, D., Postma, P., Teixeira de Mattos, J. and Cole, J.A. (Eds.), Recombinant protein production with prokaryotic and eukaryotic cells: a comparative view on host physiology. Kluwer Academic Press, Dordrecht, 2001, pp. 301-316.
Mologni, L., Salmikangas, P., Fougerousse, F., Beckmann, J.S. and Carpén, O.: « Developmental expression of myotilin, a gene mutated in limb-girdle muscular distrophy type 1A » Mech Dev 103 (2001) 121-125.
Moore, T., Hecquet, S., McLellann, A., Ville, D., Grid, D., Picard, F., Moulard, B., Asherson, P., Makoff, A.J., McCormick, D., Nashef, L., Froguel, P., Arzimanoglou, A., LeGuern, E. and Bailleul, B.: « Polymorphism analysis of JRK/JH8, the human homologue of mouse jerky, and description of a rare mutation in a case of CAE evolving to JME » Epilepsy Res 46 (2001) 157-167.
Pizzato, M., Merten, O.-W., Blair, E.D. and Takeuchi, Y.: « Development of a suspension packaging cell line for production of high titre, serum-resistant murine leukemia virus vectors » Gene Ther 8 (2001) 737-745.
Richard, I. and Beckmann, J.S.: « Molecular diagnosis of calpainopathies: methods used for detection of mutations in the CAPN3 gene implicated in limb-girdle muscular dystrophy type 2A » In: Anderson, L.V.B. and Bushby, K.M.D. (Eds.), Muscular dystrophy: Methods and protocols. Humana Press, Totowa, USA, 2001, pp. 273-288.
Sarukhan, A., Camugli, S., Gjata, B., Von Boehmer, H., Danos, O. and Jooss, K.: « Successful interference with cellular immune responses to immunogenic proteins encoded by recombinant viral vectors » J Virol 75 (2001) 269-277.
Scardino, A., Alves, P., Gross, D.A., Tourdot, S., Graff-Dubois, S., Angevin, E., Firat, H., Chouaib, S., Lemonnier, F., Nadler, L.M., Cardoso, A.A. and Kosmatopoulos, K.: « Identification of HER-2/neu immunogenic epitopes presented by renal cell carcinoma and other human epithelial tumors » Eur J Immunol 31 (2001) 3261-3270.
Soudais, C., Boutin, S. and Kremer, E.J.: « Characterization of cis-acting sequences involved in canine adenovirus packaging » Mol Ther 3 (2001) 631-640.
Soudais, C., Laplace-Builhe, C., Kissa, K. and Kremer, E.J.: « Preferential transduction of neurons by canine adenovirus vectors and their efficient retrograde transport in vivo » FASEB J 15 (2001) 2283-2285.
Spirito, F., Meneguzzi, G., Danos, O. and Mezzina, M.: « Cutaneous gene transfer and therapy: the present and the future » J Gene Med 3 (2001) 21-31.
Sylvius, N., Tesson, F., Gayet, C., Charron, P., Benaiche, A., Mangin, L., Peuchmaurd, M., Duboscq-Bidot, L., Feingold, J., Beckmann, J.S., Bouchier, C. and Komajda, M.: « A new locus for autosomal dominant dilated cardiomyopathy identified on chromosome 6q12-q14 » American Journal Of Human Genetics 68 (2001) 241-246.
Talim, B., Ognibene, A., Mattioli, E., Richard, I., Anderson, L.V. and Merlini, L.: « Normal calpain expression in genetically confirmed limb-girdle muscular dystrophy type 2A » Neurology 56 (2001) 692-3.
Tiger, C.F., Fougerousse, F., Grundstrom, G., Velling, T. and Gullberg, D.: « Alpha11beta1 integrin is a receptor for interstitial collagens involved in cell migration and collagen reorganization on mesenchymal nonmuscle cells » Dev Biol 237 (2001) 116-129.
Tores, F. and Barillot, E.: « The art of pedigree drawing: algorithmic aspects » Bioinformatics 17 (2001) 174-179.
Stockholm D, Herasse M, Marchand S, Praud C, Roudaut C, Richard I, Sebille A, Beckmann JS., « Calpain 3 mRNA expression in mice after denervation and during muscle regeneration » , Am J Physiol Cell Physiol. 2001.

Carotti M., et al. (2020) « Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery » International journal of molecular sciences 21.