Prime editing optimized RTT permits the correction of the c.8713C>T mutation in DMD gene

Duchenne muscular dystrophy is a severe debilitating genetic disease caused by different mutations in the DMD gene leading to the absence of dystrophin protein under the sarcolemma. We used CRISPR-Cas9 prime editing technology for correction of the c.8713C>T mutation in the DMD gene and tested di...

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Detalles Bibliográficos
Autores principales: Happi Mbakam, Cedric, Rousseau, Joel, Lu, Yaoyao, Bigot, Anne, Mamchaoui, Kamel, Mouly, Vincent, Tremblay, Jacques P.
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/PMC9587501/
https://www.ncbi.nlm.nih.gov/pubmed/36320324
http://dx.doi.org/10.1016/j.omtn.2022.09.022

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