Single-swap editing for the correction of common Duchenne muscular dystrophy mutations

Duchenne muscular dystrophy (DMD) is a fatal X-linked recessive disease of progressive muscle weakness and wasting caused by the absence of dystrophin protein. Current gene therapy approaches using antisense oligonucleotides require lifelong dosing and have limited efficacy in restoring dystrophin p...

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Detalles Bibliográficos
Autores principales: Chai, Andreas C., Chemello, Francesco, Li, Hui, Nishiyama, Takahiko, Chen, Kenian, Zhang, Yu, Sánchez-Ortiz, Efraín, Alomar, Adeeb, Xu, Lin, Liu, Ning, Bassel-Duby, Rhonda, Olson, Eric N.
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/PMC10192335/
https://www.ncbi.nlm.nih.gov/pubmed/37215149
http://dx.doi.org/10.1016/j.omtn.2023.04.009

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10192335/
https://www.ncbi.nlm.nih.gov/pubmed/37215149
http://dx.doi.org/10.1016/j.omtn.2023.04.009

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