Developing Bivalent Ligands to Target CUG Triplet Repeats, the Causative Agent of Myotonic Dystrophy Type 1

An expanded CUG repeat transcript (CUG(exp)) is the causative agent of myotonic dystrophy type 1 (DM1) by sequestering muscleblind-like 1 protein (MBNL1), a regulator of alternative splicing. Based on a ligand (1) that was previously reported to be active in an in vitro assay, we present the synthes...

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Main Authors: Jahromi, Amin Haghighat, Fu, Yuan, Miller, Kali A., Nguyen, Lien, Luu, Long M., Baranger, Anne M., Zimmerman, Steven C.
Formato: Artigo
Idioma:Inglês
Publicado em: 2013
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Article
Acesso em linha:https://ncbi.nlm.nih.gov/pmc/articles/PMC3925341/
https://ncbi.nlm.nih.gov/pubmed/24188018
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/jm400794z
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