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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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Detalles Bibliográficos
Autores principales:	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:	Online Artículo Texto
Lenguaje:	English
Publicado:	American Society of Gene & Cell Therapy 2020
Materias:	Article
Acceso en línea:	https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7321784/ https://www.ncbi.nlm.nih.gov/pubmed/32637445 http://dx.doi.org/10.1016/j.omtm.2020.05.024

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