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

• Main Authors: Tatiana V. Egorova (Author), Evgenia D. Zotova (Author), Denis A. Reshetov (Author), Anna V. Polikarpova (Author), Svetlana G. Vassilieva (Author), Dmitry V. Vlodavets (Author), Alexey A. Gavrilov (Author), Sergey V. Ulianov (Author), Vladimir L. Buchman (Author), Alexei V. Deykin (Author)
• Format: Book
• Published: The Company of Biologists, 2019-04-01T00:00:00Z.
• Subjects: article
• Online Access: Connect to this object online.

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...