Immunology and biotherapies Team

The immune and hematopoietic system team has two areas of expertise: one is developing new gene therapy treatments using genetically modified immune or blood cells; the other is studying unwanted immune responses in gene therapy in order to improve vectors.

The team’s researchers

The team’s projects

For their hematopoietic gene therapy projects, the team developed lentiviral vectors for clinical use, obtained preclinical data and identified several adjuvants that enhance the transduction of hematopoietic stem cells. Moreover, their studies on host / vector interactions have underlined the immunogenic nature of viral vectors. Currently, the team’s projects focus on targeting lentiviral vectors, modeling immune responses in humans and developing new approaches to immunotherapy.

Among recent publications, results for a phase I / II trial in Wiskott-Aldrich disease, for chronic X-linked granulomatosis, for Fanconi anemia, and preclinical studies for gene therapy of SCID-Artemis.

Recent publications

1. Vendomele J, Dehmani S, Khebizi Q, Galy A, and Fisson S (2018). Subretinal Injection of HY Peptides Induces Systemic Antigen-Specific Inhibition of Effector CD4(+) and CD8(+) T-Cell Responses. Frontiers in Immunology 9, 504.
   
   
2. Boudeffa D, Bertin B, Biek A, Mormin M, Leseigneur F, Galy A, and Merten OW (2019). Toward a Scalable Purification Protocol of GaLV-TR-Pseudotyped Lentiviral Vectors. Human Gene Therapy Methods 30, 153-171.
   
   
3. Charrier S, Lagresle-Peyrou C, Poletti V, Rothe M, Cedrone G, Gjata B, Mavilio F, Fischer A, Schambach A, de Villartay JP, Cavazzana M, Hacein-Bey-Abina S, and Galy A (2019). Biosafety Studies of a Clinically Applicable Lentiviral Vector for the Gene Therapy of Artemis-SCID. Molecular Therapy Methods & Clinical Development 15, 232-245.
   
   
4. Río P, Navarro S, Wang W, Sánchez-Domínguez R, Pujol RM, Segovia JC, Bogliolo M, Merino E, Wu N, Salgado R, Lamana ML, Yañez RM, Casado JA, Giménez Y, Román-Rodríguez FJ, Álvarez L, Alberquilla O, Raimbault A, Guenechea G, Lozano ML, Cerrato L, Hernando M, Gálvez E, Hladun R, Giralt I, Barquinero J, Galy A, García de Andoín N, López R, Catalá A, Schwartz JD, Surrallés J, Soulier J, Schmidt M, Díaz de Heredia C, Sevilla J, and Bueren JA (2019). Successful engraftment of gene-corrected hematopoietic stem cells in non-conditioned patients with Fanconi anemia. Nature Medicine 25, 1396-1401.
   
   
5. Kohn DB, Booth C, Kang EM, Pai SY, Shaw KL, Santilli G, Armant M, Buckland KF, Choi U, De Ravin SS, Dorsey MJ, Kuo CY, Leon-Rico D, Rivat C, Izotova N, Gilmour K, Snell K, Dip JX, Darwish J, Morris EC, Terrazas D, Wang LD, Bauser CA, Paprotka T, Kuhns DB, Gregg J, Raymond HE, Everett JK, Honnet G, Biasco L, Newburger PE, Bushman FD, Grez M, Gaspar HB, Williams DA, Malech HL, Galy A, and Thrasher AJ (2020). Lentiviral gene therapy for X-linked chronic granulomatous disease. Nat Med 26 (2), 200-206.

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