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...

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Detalhes bibliográficos
Publicado no:Mol Ther Methods Clin Dev
Main Authors: Ikeda, Miki, Taniguchi-Ikeda, Mariko, Kato, Takema, Shinkai, Yasuko, Tanaka, Sonoko, Hagiwara, Hiroki, Sasaki, Naomichi, Masaki, Toshihiro, Matsumura, Kiichiro, Sonoo, Masahiro, Kurahashi, Hiroki, Saito, Fumiaki
Formato: Artigo
Idioma:Inglês
Publicado em: American Society of Gene & Cell Therapy 2020
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC7321784/
https://ncbi.nlm.nih.gov/pubmed/32637445
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.omtm.2020.05.024
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