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Rational Design of Bioactive, Modularly Assembled Aminoglycosides Targeting the RNA that Causes Myotonic Dystrophy Type 1

Myotonic dystrophy type 1 (DM1) is caused when an expanded r(CUG) repeat (r(CUG)(exp)) binds the RNA splicing regulator muscleblind-like 1 protein (MBNL1) as well as other proteins. Previously, we reported that modularly assembled small molecules displaying a 6′-N-5-hexynoate kanamycin A RNA-binding...

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Bibliographic Details
Main Authors: Childs-Disney, Jessica L., Parkesh, Raman, Nakamori, Masayuki, Thornton, Charles A., Disney, Matthew D.
Format: Artigo
Language:Inglês
Published: 2012
Subjects:
Online Access:https://ncbi.nlm.nih.gov/pmc/articles/PMC3528830/
https://ncbi.nlm.nih.gov/pubmed/23130637
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1021/cb3001606
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