Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach

Unexpected Mutations by CRISPR-Cas9 CTG Repeat Excision in Myotonic Dystrophy and Use of CRISPR Interference as an Alternative Approach

Myotonic dystrophy type 1 is the most common type of adult-onset muscular dystrophy. This is an autosomal dominant disorder and caused by the expansion of the CTG repeat in the 3′ untranslated region of the dystrophia myotonica protein kinase (DMPK) gene. Messenger RNAs containing these expanded rep...

Full description

Bibliographic Details
Main Authors: Miki Ikeda, Mariko Taniguchi-Ikeda, Takema Kato, Yasuko Shinkai, Sonoko Tanaka, Hiroki Hagiwara, Naomichi Sasaki, Toshihiro Masaki, Kiichiro Matsumura, Masahiro Sonoo, Hiroki Kurahashi, Fumiaki Saito
Format: Article
Language:English
Published: Elsevier 2020-09-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
CRISPR-Cas9
genome editing
myotonic dystrophy
DMPK
RNA foci
off-target
Online Access:http://www.sciencedirect.com/science/article/pii/S232905012030111X

Internet

http://www.sciencedirect.com/science/article/pii/S232905012030111X

Similar Items