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

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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Bibliographic Details
Main Authors: Tatiana V. Egorova, Evgenia D. Zotova, Denis A. Reshetov, Anna V. Polikarpova, Svetlana G. Vassilieva, Dmitry V. Vlodavets, Alexey A. Gavrilov, Sergey V. Ulianov, Vladimir L. Buchman, Alexei V. Deykin
Format: Article
Language:English
Published: The Company of Biologists 2019-04-01
Series:Disease Models & Mechanisms
Subjects:
Exon skipping
Muscle degeneration
New deletion
Genome editing
DMD
Mouse model
Online Access:http://dmm.biologists.org/content/12/4/dmm037655

Internet

http://dmm.biologists.org/content/12/4/dmm037655

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