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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Detalhes bibliográficos
Publicado no:Dis Model Mech
Principais autores: 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: Artigo
Idioma:Inglês
Publicado em: The Company of Biologists Ltd 2019
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Resource Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC6505476/
https://ncbi.nlm.nih.gov/pubmed/31028078
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1242/dmm.037655
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