A humanized knockin mouse model of Duchenne muscular dystrophy and its correction by CRISPR-Cas9 therapeutic gene editing

Duchenne muscular dystrophy (DMD) is a lethal neuromuscular disease caused by mutations in the X-linked dystrophin (DMD) gene. Exon deletions flanking exon 51, which disrupt the dystrophin open reading frame (ORF), represent one of the most common types of human DMD mutations. Previously, we used cl...

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Detalles Bibliográficos
Autores principales: Zhang, Yu, Li, Hui, Nishiyama, Takahiko, McAnally, John R., Sanchez-Ortiz, Efrain, Huang, Jian, Mammen, Pradeep P.A., Bassel-Duby, Rhonda, Olson, Eric N.
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/PMC9398917/
https://www.ncbi.nlm.nih.gov/pubmed/36035749
http://dx.doi.org/10.1016/j.omtn.2022.07.024

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC9398917/
https://www.ncbi.nlm.nih.gov/pubmed/36035749
http://dx.doi.org/10.1016/j.omtn.2022.07.024

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