A consolidated AAV system for single-cut CRISPR correction of a common Duchenne muscular dystrophy mutation

Duchenne muscular dystrophy (DMD), caused by mutations in the X-linked dystrophin gene, is a lethal neuromuscular disease. Correction of DMD mutations in animal models has been achieved by CRISPR/Cas9 genome editing using Streptococcus pyogenes Cas9 (SpCas9) delivered by adeno-associated virus (AAV)...

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Detalles Bibliográficos
Autores principales: Zhang, Yu, Nishiyama, Takahiko, Li, Hui, Huang, Jian, Atmanli, Ayhan, Sanchez-Ortiz, Efrain, Wang, Zhaoning, Mireault, Alex A., Mammen, Pradeep P.A., Bassel-Duby, Rhonda, Olson, Eric N.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8397837/
https://www.ncbi.nlm.nih.gov/pubmed/34485599
http://dx.doi.org/10.1016/j.omtm.2021.05.014

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