Design of a Bioactive Small Molecule That Targets the Myotonic Dystrophy Type 1 RNA via an RNA Motif–Ligand Database and Chemical Similarity Searching

Design of a Bioactive Small Molecule That Targets the Myotonic Dystrophy Type 1 RNA via an RNA Motif–Ligand Database and Chemical Similarity Searching

Myotonic dystrophy type 1 (DM1) is a triplet repeating disorder caused by expanded CTG repeats in the 3′-untranslated region of the dystrophia myotonica protein kinase (DMPK) gene. The transcribed repeats fold into an RNA hairpin with multiple copies of a 5′CUG/3′GUC motif that binds the RNA splicin...

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Bibliographic Details
Main Authors: Parkesh, Raman, Childs-Disney, Jessica L., Nakamori, Masayuki, Kumar, Amit, Wang, Thomas, Hoskins, Jason, Thornton, Charles A., Disney, Matthew D., Housman, David E, Wang, Eric T, Tran, Tuan, M. Eng (Tuan Minh) Massachusetts Institute of Technology
Other Authors: Massachusetts Institute of Technology. Department of Biology
Format: Article
Language:en_US
Published: American Chemical Society (ACS) 2014
Online Access:http://hdl.handle.net/1721.1/84533
https://orcid.org/0000-0001-5016-0756

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