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Small Molecules that Target the Toxic RNA in Myotonic Dystrophy Type 2
Myotonic dystrophy type 2 is caused by an expansion of CCTG repeats in the zinc-finger protein gene (ZNF9). Transcribed CCUG repeats sequester MBNL1, an important alternative splicing regulator, preventing its normal function, leading to the disease phenotype. We describe a series of ligands that di...
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| Publicado no: | ChemMedChem |
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| Main Authors: | , , |
| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado em: |
2014
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| Assuntos: | |
| Acesso em linha: | https://ncbi.nlm.nih.gov/pmc/articles/PMC4320974/ https://ncbi.nlm.nih.gov/pubmed/24938413 https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1002/cmdc.201402095 |
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