Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy

Duchenne muscular dystrophy (DMD) is a devastating muscle-wasting disease that arises due to the loss of dystrophin expression, leading to progressive loss of motor and cardiorespiratory function. Four exon-skipping approaches using antisense phosphorodiamidate morpholino oligomers (PMOs) have been...

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Detalles Bibliográficos
Autores principales: Gushchina, Liubov V., Vetter, Tatyana A., Frair, Emma C., Bradley, Adrienne J., Grounds, Kelly M., Lay, Jacob W., Huang, Nianyuan, Suhaiba, Aisha, Schnell, Frederick J., Hanson, Gunnar, Simmons, Tabatha R., Wein, Nicolas, Flanigan, Kevin M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9678653/
https://www.ncbi.nlm.nih.gov/pubmed/36420217
http://dx.doi.org/10.1016/j.omtn.2022.10.025

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9678653/
https://www.ncbi.nlm.nih.gov/pubmed/36420217
http://dx.doi.org/10.1016/j.omtn.2022.10.025

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