Lack of toxicity of alpha-sarcoglycan overexpression supports clinical gene transfer trial in LGMD2D

Scritto il . Pubblicato in Terapia genica.

https://pubmed.ncbi.nlm.nih.gov/18525034/

Rodino-Klapac LR, Lee JS, Mulligan RC, Clark KR, Mendell JR.

Neurology. 2008 Jul 22;71(4):240-7. Epub 2008 Jun 4.