Design of novel small molecule base-pair recognizers of toxic CUG RNA transcripts characteristics of DM1

Myotonic Dystrophy type 1 (DM1) is an incurable neuromuscular disorder caused by toxic DMPK transcripts that carry CUG repeat expansions in the 3′ untranslated region (3′UTR). The intrinsic complexity and lack of crystallographic data makes noncoding RNA regions challenging targets to study in the f...

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Main Authors: Raul Ondono, Ángel Lirio, Carlos Elvira, Elena Álvarez-Marimon, Claudia Provenzano, Beatrice Cardinali, Manuel Pérez-Alonso, Alex Perálvarez-Marín, José I. Borrell, Germana Falcone, Roger Estrada-Tejedor
Format: Article
Language:English
Published: Elsevier 2021-01-01
Series:Computational and Structural Biotechnology Journal
Subjects:
Online Access:http://www.sciencedirect.com/science/article/pii/S2001037020305201