Development of versatile allele-specific siRNAs able to silence all the dominant dynamin 2 mutations

Dominant centronuclear myopathy (CNM) is a rare form of congenital myopathy associated with a wide clinical spectrum, from severe neonatal to milder adult forms. There is no available treatment for this disease due to heterozygous mutations in the DNM2 gene encoding Dynamin 2 (DNM2). Dominant DNM2 m...

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Detalles Bibliográficos
Autores principales: Dudhal, Swati, Mekzine, Lylia, Prudhon, Bernard, Soocheta, Karishma, Cadot, Bruno, Mamchaoui, Kamel, Trochet, Delphine, 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/PMC9439966/
https://www.ncbi.nlm.nih.gov/pubmed/36090755
http://dx.doi.org/10.1016/j.omtn.2022.08.016

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