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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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| Main Authors: | , , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
Nature Publishing Group
2014
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| Assuntos: | |
| Acesso em linha: | 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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