Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model

Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by mutations in the DMD gene that prevent the expression of a functional dystrophin protein. Exon duplications represent 6%–11% of mutations, and duplications of exon 2 (Dup2) are the most common (∼11%) of duplication mutatio...

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Detalles Bibliográficos
Autores principales: Gushchina, Liubov V., Bradley, Adrienne J., Vetter, Tatyana A., Lay, Jacob W., Rohan, Natalie L., Frair, Emma C., Wein, Nicolas, Flanigan, Kevin M.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2023
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10679948/
https://www.ncbi.nlm.nih.gov/pubmed/38027058
http://dx.doi.org/10.1016/j.omtm.2023.101144

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10679948/
https://www.ncbi.nlm.nih.gov/pubmed/38027058
http://dx.doi.org/10.1016/j.omtm.2023.101144

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