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

International audienc

γ-sarcoglycan and dystrophin mutation spectrum in an Algerian cohort

International audienceWe report the genetic analysis of a large series of 76 Algerian patients from 65 unrelated families who presented with early onset severe muscular dystrophy and a clinical phenotype resembling limb-girdle muscular dystrophy type 2C