Title: | Titin splicing regulates cardiotoxicity associated with calpain 3 gene therapy for limb-girdle muscular dystrophy type 2A |
Journal : | Science translational medicine, 11, 520 |
Authors: | Lostal W ; Roudaut C ; Faivre M ; Charton K ; Suel L ; Bourg N ; Best H ; Smith JE ; Gohlke J ; Corre G ; Li X ; Elbeck Z ; Knöll R ; Deschamps JY ; Granzier H ; Richard I |
Material Type: | Article |
Publisher: | United States, 11/2019 |
Languages: | English |
Keywords : | adenoassociated vectors ; adverse side effects ; calpain 3 ; Calpain-3-related limb-girdle muscular dystrophy R1 ; calpainopathy ; calpains ; gene therapy ; limb girdle muscular dystrophy ; mouse ; neuromuscular disease ; preclinical trials ; Primates |
Pubmed / DOI : | Pubmed : 31776291 / DOI : 10.1126/scitranslmed.aat6072 |
N° Profil MNM : | 2019121 |
Link for e-copy: | http://www.ncbi.nlm.nih.gov/pubmed/31776291 |
See also : |