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.

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Autores principales: 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
Formato: article
Lenguaje:EN
Publicado: Nature Portfolio 2018
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Science
Q
Acceso en línea:https://doaj.org/article/cc1752fb9d394b0fb636e7fca670afcc
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