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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Vydáno v:Mol Ther Methods Clin Dev
Hlavní autoři: Simmons, Tabatha R., Vetter, Tatyana A., Huang, Nianyuan, Vulin-Chaffiol, Adeline, Wein, Nicolas, Flanigan, Kevin M.
Médium: Artigo
Jazyk:Inglês
Vydáno: American Society of Gene & Cell Therapy 2021
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Original Article
On-line přístup: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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