Authors: William Lostal, Carinne Roudaut, Marine Faivre, Karine Charton, Laurence Suel, Nathalie Bourg, Heather Best, John Edward Smith, Jochen Gohlke, Guillaume Corre, Xidan Li, Zaher Elbeck, Ralph Knöll, Jack-Yves Deschamps, Henk Granzier, Isabelle Richard
Published: 2019-11-28
DOI: 10.1126/scitranslmed.aat6072
Source: Full article
Preclinical studies demonstrate the potential of calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A while avoiding cardiotoxicity.