AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression

Evelyne Gicquel (1) , Natacha Maizonnier (1) , Steven Foltz (2) , William Martin , Nathalie Bourg (1) , Fedor Svinartchouk (1) , Karine Charton (1) , Aaron M. Beedle (2) , Isabelle Richard (1)

1 INTEGRARE - Approches génétiques intégrées et nouvelles thérapies pour les maladies rares
2 University of Georgia [USA]

Keywords: Muscle Dystrophy; LGMD; FKRP

Domains: Human health and pathology; Biotechnology

Evelyne Gicquel, Natacha Maizonnier, Steven Foltz, William Martin, Nathalie Bourg, et al.. AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression. Human Molecular Genetics, 2017, 26 (10), pp.1952-1965. ⟨10.1093/hmg/ddx066⟩. ⟨hal-02177575⟩