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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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Detalles Bibliográficos
Publicado en:ChemMedChem
Main Authors: Nguyen, Lien, Lee, JuYeon, Zimmerman, Steven C.
Formato: Artigo
Idioma:Inglês
Publicado: 2014
Assuntos:
Acceso en liña: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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