Treatment of Type 1 Myotonic Dystrophy by Engineering Site-specific RNA Endonucleases that Target (CUG)(n) Repeats

Myotonic dystrophy type 1 (DM1) is caused by the expansion of (CTG)(n) in the 3′ untranslated region of the dystrophia myotonica-protein kinase (DMPK) gene, which is transcribed as (CUG)(n) repeats that accumulate in the nucleus. The RNA repeats specifically sequester or change the expression levels...

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Autori principali: Zhang, Wenjing, Wang, Yang, Dong, Shuyun, Choudhury, Rajarshi, Jin, Yongfeng, Wang, Zefeng
Natura: Artigo
Lingua:Inglês
Pubblicazione: Nature Publishing Group 2014
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Original Article
Accesso online:https://ncbi.nlm.nih.gov/pmc/articles/PMC3916045/
https://ncbi.nlm.nih.gov/pubmed/24196578
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1038/mt.2013.251
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