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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Publicat a:Nat Biomed Eng
Autors principals: 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.
Format: Artigo
Idioma:Inglês
Publicat: 2020
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Article
Accés en línia: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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