Sustained alpha-sarcoglycan gene expression after gene transfer in limb-girdle muscular dystrophy, type 2D.
Mendell JR, Rodino-Klapac LR, Rosales XQ, Coley BD, Galloway G, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Taylor LE, Flanigan KM, Gastier-Foster JM, Astbury C, Kota J, Sahenk Z, Walker CM, Clark KR.
Mendell JR, et al. Among authors: gastier foster jm.
Ann Neurol. 2010 Nov;68(5):629-38. doi: 10.1002/ana.22251.
Ann Neurol. 2010.
PMID: 21031578
Free PMC article.
Clinical Trial.
INTERPRETATION: The findings of this gene replacement study in LGMD2D subjects have important implications not previously demonstrated in muscular dystrophy. Long-term, sustainable gene expression of alpha-sarcoglycan was observed following gene transfer mediated by AAV. . …
INTERPRETATION: The findings of this gene replacement study in LGMD2D subjects have important implications not previously demonstrated in mu …