Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes

Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes

Spinal muscular atrophy (SMA) is an autosomal recessive disorder with no present cure. Here the authors perform an in vitro screening leading to the identification of a small molecule that alters the conformational dynamics of the TSL2 RNA structure and acts as a modulator of SMN exon 7 splicing.

Enregistré dans:
Détails bibliographiques
Auteurs principaux: Amparo Garcia-Lopez, Francesca Tessaro, Hendrik R. A. Jonker, Anna Wacker, Christian Richter, Arnaud Comte, Nikolaos Berntenis, Roland Schmucki, Klas Hatje, Olivier Petermann, Gianpaolo Chiriano, Remo Perozzo, Daniel Sciarra, Piotr Konieczny, Ignacio Faustino, Guy Fournet, Modesto Orozco, Ruben Artero, Friedrich Metzger, Martin Ebeling, Peter Goekjian, Benoît Joseph, Harald Schwalbe, Leonardo Scapozza
Format: article
Langue:EN
Publié: Nature Portfolio 2018
Sujets:
Science
Q
Accès en ligne:https://doaj.org/article/cc1752fb9d394b0fb636e7fca670afcc
Tags: Ajouter un tag
Pas de tags, Soyez le premier à ajouter un tag!

Internet

https://doaj.org/article/cc1752fb9d394b0fb636e7fca670afcc

Documents similaires