Cas9-induced single cut enables highly efficient and template-free repair of a muscular dystrophy causing founder mutation

With thousands of patients worldwide, CAPN3 c.550delA is the most frequent mutation causing severe, progressive, and untreatable limb girdle muscular dystrophy. We aimed to genetically correct this founder mutation in primary human muscle stem cells. We designed editing strategies providing CRISPR-C...

Descripción completa

Detalles Bibliográficos
Autores principales: Müthel, Stefanie, Marg, Andreas, Ignak, Busem, Kieshauer, Janine, Escobar, Helena, Stadelmann, Christian, Spuler, Simone
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/PMC9972404/
https://www.ncbi.nlm.nih.gov/pubmed/36865086
http://dx.doi.org/10.1016/j.omtn.2023.02.005

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9972404/
https://www.ncbi.nlm.nih.gov/pubmed/36865086
http://dx.doi.org/10.1016/j.omtn.2023.02.005

Ejemplares similares