Selective inhibition of MBNL1–CCUG interaction by small molecules toward potential therapeutic agents for myotonic dystrophy type 2 (DM2)(†)

Myotonic dystrophy type 2 (DM2) is an incurable neuromuscular disease caused by expanded CCUG repeats that may exhibit toxicity by sequestering the splicing regulator MBNL1. A series of triaminotriazine- and triaminopyrimidine-based small molecules (ligands 1–3) were designed, synthesized and tested...

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Bibliographic Details
Main Authors: Wong, Chun-Ho, Fu, Yuan, Ramisetty, Sreenivasa Rao, Baranger, Anne M., Zimmerman, Steven C.
Format: Artigo
Language:Inglês
Published: Oxford University Press 2011
Subjects:
RNA
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3203617/
https://ncbi.nlm.nih.gov/pubmed/21768123
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1093/nar/gkr415
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