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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Udgivet i:Nat Biomed Eng
Main Authors: 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
Sprog:Inglês
Udgivet: 2020
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Article
Online adgang: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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