AAV-mediated transfer of FKRP shows therapeutic efficacy in a murine model but requires control of gene expression
Gicquel, Evelyne, Maizonnier, Natacha, Foltz, Steven J., Martin, William J., Bourg, Nathalie, Svinartchouk, Fedor, Charton, Karine, Beedle, Aaron M., Richard, IsabelleLanguage:
english
Journal:
Human Molecular Genetics
DOI:
10.1093/hmg/ddx066
Date:
March, 2017
File:
PDF, 1.20 MB
english, 2017