CRISPR/Cas9-generated mouse model of Duchenne muscular dystrophy recapitulating a newly identified large 430 kb deletion in the human DMD gene

Exon skipping is a promising strategy for Duchenne muscular dystrophy (DMD) disease-modifying therapy. To make this approach safe, ensuring that excluding one or more exons will restore the reading frame and that the resulting protein will retain critical functions of the full-length dystrophin prot...

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Detalles Bibliográficos
Autores principales: Egorova, Tatiana V., Zotova, Evgenia D., Reshetov, Denis A., Polikarpova, Anna V., Vassilieva, Svetlana G., Vlodavets, Dmitry V., Gavrilov, Alexey A., Ulianov, Sergey V., Buchman, Vladimir L., Deykin, Alexei V.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: The Company of Biologists Ltd 2019
Materias:
Resource Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC6505476/
https://www.ncbi.nlm.nih.gov/pubmed/31028078
http://dx.doi.org/10.1242/dmm.037655

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