Long-term maintenance of dystrophin expression and resistance to injury of skeletal muscle in gene edited DMD mice

Duchenne muscular dystrophy (DMD) is a lethal muscle disease caused by mutations in the dystrophin gene. CRISPR/Cas9 genome editing has been used to correct DMD mutations in animal models at young ages. However, the longevity and durability of CRISPR/Cas9 editing remained to be determined. To addres...

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Detalles Bibliográficos
Autores principales: Karri, Dileep R., Zhang, Yu, Chemello, Francesco, Min, Yi-Li, Huang, Jian, Kim, Jiwoong, Mammen, Pradeep P.A., Xu, Lin, Liu, Ning, 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/PMC8956962/
https://www.ncbi.nlm.nih.gov/pubmed/35402069
http://dx.doi.org/10.1016/j.omtn.2022.03.004

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