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Antisense Oligonucleotides: Rising Stars in Eliminating RNA Toxicity in Myotonic Dystrophy
Myotonic dystrophy (DM) is a dominantly inherited, multisystemic disease caused by expanded CTG (type 1, DM1) or CCTG (type 2, DM2) repeats in untranslated regions of the mutated genes. Pathogenesis results from expression of RNAs from the mutated alleles that are toxic because of the expanded CUG o...
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| Main Authors: | , |
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| Format: | Artigo |
| Language: | Inglês |
| Published: |
Mary Ann Liebert, Inc.
2013
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| Subjects: | |
| Online Access: | https://ncbi.nlm.nih.gov/pmc/articles/PMC3655630/ https://ncbi.nlm.nih.gov/pubmed/23252746 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1089/hum.2012.212 |
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