Limb-girdle muscular dystrophy type 2D gene therapy restores alpha-sarcoglycan and associated proteins

https://pubmed.ncbi.nlm.nih.gov/19798725/

Mendell JR, Rodino-Klapac LR, Rosales-Quintero X, Kota J, Coley BD, Galloway G, Craenen JM, Lewis S, Malik V, Shilling C, Byrne BJ, Conlon T, Campbell KJ, Bremer WG, Viollet L, Walker CM, Sahenk Z, Clark KR.

Ann Neurol. 2009 Sep;66(3):290-7.

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