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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Detalles Bibliográficos
Autores principales: Simmons, Tabatha R., Vetter, Tatyana A., Huang, Nianyuan, Vulin-Chaffiol, Adeline, Wein, Nicolas, Flanigan, Kevin M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8047432/
https://www.ncbi.nlm.nih.gov/pubmed/33898631
http://dx.doi.org/10.1016/j.omtm.2021.03.014

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8047432/
https://www.ncbi.nlm.nih.gov/pubmed/33898631
http://dx.doi.org/10.1016/j.omtm.2021.03.014

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