Benefits of therapy by dynamin-2-mutant-specific silencing are maintained with time in a mouse model of dominant centronuclear myopathy

Dominant dynamin 2 (DNM2) mutations are responsible for the autosomal dominant centronuclear myopathy (AD-CNM), a rare progressive neuromuscular disorder ranging from severe neonatal to mild adult forms. We previously demonstrated that mutant-specific RNA interference is an efficient therapeutic str...

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Detalles Bibliográficos
Autores principales: Trochet, Delphine, Prudhon, Bernard, Mekzine, Lylia, Lemaitre, Mégane, Beuvin, Maud, Julien, Laura, Benkhelifa-Ziyyat, Sofia, Bui, Mai Thao, Romero, Norma, Bitoun, Marc
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/PMC8889367/
https://www.ncbi.nlm.nih.gov/pubmed/35282416
http://dx.doi.org/10.1016/j.omtn.2022.02.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8889367/
https://www.ncbi.nlm.nih.gov/pubmed/35282416
http://dx.doi.org/10.1016/j.omtn.2022.02.009

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