Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse

Duchenne muscular dystrophy (DMD) is typically caused by mutations that disrupt the DMD reading frame, but nonsense mutations in the 5′ part of the gene induce utilization of an internal ribosomal entry site (IRES) in exon 5, driving expression of a highly functional N-truncated dystrophin. We have...

Descripción completa

Detalles Bibliográficos
Autores principales: Wein, Nicolas, Vetter, Tatyana A., Vulin, Adeline, Simmons, Tabatha R., Frair, Emma C., Bradley, Adrienne J., Gushchina, Liubov V., Almeida, Camila F., Huang, Nianyuan, Lesman, Daniel, Rajakumar, Dhanarajan, Weiss, Robert B., 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/PMC9356240/
https://www.ncbi.nlm.nih.gov/pubmed/35949298
http://dx.doi.org/10.1016/j.omtm.2022.07.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9356240/
https://www.ncbi.nlm.nih.gov/pubmed/35949298
http://dx.doi.org/10.1016/j.omtm.2022.07.005

Ejemplares similares