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Rational design of ligands targeting triplet repeating transcripts that cause RNA dominant disease: Application to myotonic muscular dystrophy type 1 and spinocerebellar ataxia type 3

Herein, we describe the design of high affinity ligands that bind expanded rCUG- and rCAG-repeat RNAs expressed in myotonic dystrophy and spinocerebellar ataxia. These ligands also inhibit, with nanomolar IC(50)'s, the formation of RNA-protein complexes that are implicated in both disorders. Th...

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Detalhes bibliográficos
Main Authors: Pushechnikov, Alexei, Lee, Melissa M., Childs-Disney, Jessica L., Sobczak, Krzysztof, French, Jonathan M., Thornton, Charles A., Disney, Matthew D.
Formato: Artigo
Idioma:Inglês
Publicado em: 2009
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Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC2731475/
https://ncbi.nlm.nih.gov/pubmed/19552411
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/ja9020149
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