The sustained expression of Cas9 targeting toxic RNAs reverses disease phenotypes in mouse models of myotonic dystrophy type 1

Myotonic dystrophy type I (DM1) is a multisystemic autosomal-dominant inherited human disorder that is caused by CTG microsatellite repeat expansions (MREs) in the 3′ untranslated region of DMPK. Toxic RNAs expressed from such repetitive sequences can be eliminated using CRISPR-mediated RNA targetin...

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Detaylı Bibliyografya
Yayımlandı:Nat Biomed Eng
Asıl Yazarlar: Batra, Ranjan, Nelles, David A., Roth, Daniela M., Krach, Florian, Nutter, Curtis A., Tadokoro, Takahiro, Thomas, James D., Sznajder, Łukasz J., Blue, Steven M., Gutierrez, Haydee L., Liu, Patrick, Aigner, Stefan, Platoshyn, Oleksandr, Miyanohara, Atsushi, Marsala, Martin, Swanson, Maurice S., Yeo, Gene W.
Materyal Türü: Artigo
Dil:Inglês
Baskı/Yayın Bilgisi: 2020
Konular:
Article
Online Erişim:https://ncbi.nlm.nih.gov/pmc/articles/PMC8241012/
https://ncbi.nlm.nih.gov/pubmed/32929188
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/s41551-020-00607-7
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