377. AAV-Mediated Transfer of FKRP Shows Therapeutic Efficacy in a Murine Model of Limb-Girdle Muscular Dystrophy Type 2i, but Requires Tight Control of Gene Expression
Gicquel, Evelyne, Maizonnier, Natacha, Foltz, Steven J., Martin, William, Bourg, Nathalie, Svinartchouk, Fedor, Charton, Karine, Beedle, Aaron, Richard, IsabelleVolume:
24
Language:
english
Journal:
Molecular Therapy
DOI:
10.1016/s1525-0016(16)33186-0
Date:
May, 2016
File:
PDF, 337 KB
english, 2016