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...

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Pubblicato in:Hum Gene Ther
Autori principali: Potter, Rachael A., Griffin, Danielle A., Heller, Kristin N., Peterson, Ellyn L., Clark, Emma K., Mendell, Jerry R., Rodino-Klapac, Louise R.
Natura: Artigo
Lingua:Inglês
Pubblicazione: Mary Ann Liebert, Inc., publishers 2021
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Research Articles
Accesso online: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
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