Pre-clinical dose-escalation studies establish a therapeutic range for U7snRNA-mediated DMD exon 2 skipping

Duchenne muscular dystrophy (DMD) is an X-linked progressive disease characterized by loss of dystrophin protein that typically results from truncating mutations in the DMD gene. Current exon-skipping therapies have sought to treat deletion mutations that abolish an open reading frame (ORF) by skipp...

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Bibliografiske detaljer
Udgivet i:Mol Ther Methods Clin Dev
Main Authors: Simmons, Tabatha R., Vetter, Tatyana A., Huang, Nianyuan, Vulin-Chaffiol, Adeline, Wein, Nicolas, Flanigan, Kevin M.
Format: Artigo
Sprog:Inglês
Udgivet: American Society of Gene & Cell Therapy 2021
Fag:
Original Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC8047432/
https://ncbi.nlm.nih.gov/pubmed/33898631
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2021.03.014
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