Dose-Escalation Study of Systemically Delivered rAAVrh74.MHCK7.micro-dystrophin in the mdx Mouse Model of Duchenne Muscular Dystrophy

Duchenne muscular dystrophy (DMD) is a rare, X-linked, fatal, degenerative neuromuscular disease caused by mutations in the DMD gene. More than 2,000 mutations of the DMD gene are responsible for progressive loss of muscle strength, loss of ambulation, and generally respiratory and cardiac failure b...

Description complète

Enregistré dans:
Détails bibliographiques
Publié dans:Hum Gene Ther
Auteurs principaux: Potter, Rachael A., Griffin, Danielle A., Heller, Kristin N., Peterson, Ellyn L., Clark, Emma K., Mendell, Jerry R., Rodino-Klapac, Louise R.
Format: Artigo
Langue:Inglês
Publié: Mary Ann Liebert, Inc., publishers 2021
Sujets:
Research Articles
Accès en ligne:https://ncbi.nlm.nih.gov/pmc/articles/PMC8063270/
https://ncbi.nlm.nih.gov/pubmed/33397205
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2019.255
Tags: Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!

Documents similaires