The aim of this study was to attain long-lasting alpha-sarcoglycan gene expression in limb-girdle muscular dystrophy, type 2D (LGMD2D) subjects mediated by adeno-associated virus (AAV) gene transfer under control of a muscle specific promoter (tMCK).
Journal Article,
Research Support, U.S. Gov't, P.H.S.,
Randomized Controlled Trial,
Research Support, Non-U.S. Gov't,
Research Support, N.I.H., Extramural