Impeding transcription of expanded microsatellite repeats by deactivated Cas9

Transcription of expanded microsatellite repeats is associated with multiple human diseases, including myotonic dystrophy, Fuchs' endothelial corneal dystrophy, and C9orf72-ALS/FTD. Reducing production of RNA and proteins arising from these expanded loci holds therapeutic benefit. Here, we test...

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Udgivet i:Mol Cell
Main Authors: Pinto, Belinda S., Saxena, Tanvi, Oliveira, Ruan, Méndez-Gómez, Héctor R., Cleary, John D., Denes, Lance T., McConnell, Ona, Arboleda, Juan, Xia, Guangbin, Swanson, Maurice S., Wang, Eric T.
Format: Artigo
Sprog:Inglês
Udgivet: 2017
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Article
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6013302/
https://ncbi.nlm.nih.gov/pubmed/29056323
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1016/j.molcel.2017.09.033
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