Elimination of Toxic Microsatellite Repeat Expansion RNA by RNA-Targeting Cas9

Microsatellite repeat expansions in DNA produce pathogenic RNA species that cause dominantly inherited diseases such as myotonic dystrophy type 1 and 2 (DM1/2), Huntington’s disease, and C9orf72-linked amyotrophic lateral sclerosis (C9-ALS). Means to target these repetitive RNAs are required for dia...

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Pubblicato in:Cell
Autori principali: Batra, Ranjan, Nelles, David A., Pirie, Elaine, Blue, Steven M., Marina, Ryan J., Wang, Harrison, Chaim, Isaac A., Thomas, James D., Zhang, Nigel, Nguyen, Vu, Aigner, Stefan, Markmiller, Sebastian, Xia, Guangbin, Corbett, Kevin D., Swanson, Maurice S., Yeo, Gene W.
Natura: Artigo
Lingua:Inglês
Pubblicazione: 2017
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Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC5873302/
https://ncbi.nlm.nih.gov/pubmed/28803727
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.cell.2017.07.010
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