Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes

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Authors: 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, BenoƮt Joseph, Harald Schwalbe, Leonardo Sca

Journal title: Nature Communications

Journal number: 9/1

Journal publisher: Nature Publishing Group

Published year: 2018

DOI identifier: 10.1038/s41467-018-04110-1

ISSN: 2041-1723