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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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| Autors principals: | , , , , , , |
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| Format: | Artigo |
| Idioma: | Inglês |
| Publicat: |
2009
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| Matèries: | |
| Accés en línia: | 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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