2020
Carotti M., et al. (2020) « Combined Use of CFTR Correctors in LGMD2D Myotubes Improves Sarcoglycan Complex Recovery » Int J Mol Sci 21(5), 1813.
Ronzitti G., et al. (2020)
« Human Immune Responses to Adeno-Associated Virus (AAV) Vectors »
Front Immunol 11, 670.
Barbon E., et al. (2020)
« Single-domain antibodies targeting antithrombin reduce bleeding in hemophilic mice with or without inhibitors »
EMBO molecular medicine 12, e11298.
Dupont JB., et al. (2020)
« AAV-Mediated Gene Transfer Restores a Normal Muscle Transcriptome in a Canine Model of X-Linked Myotubular Myopathy »
Mol Ther 28(2), 382-393.
Six E., et al. (2020)
« Clonal tracking in gene therapy patients reveals a diversity of human hematopoietic differentiation programs »
Blood 135(15), 1219-1231.
