Systemic AAV-Mediated β-Sarcoglycan Delivery Targeting Cardiac and Skeletal Muscle Ameliorates Histological and Functional Deficits in LGMD2E Mice

Scritto il . Pubblicato in Terapia genica.

Pozsgai ER1, Griffin DA2, Heller KN2, Mendell JR3, Rodino-Klapac LR4.

https://pubmed.ncbi.nlm.nih.gov/28284983/