Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes
Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes
About this item
Full title
Author / Creator
Garcia-Lopez, Amparo , Tessaro, Francesca , Jonker, Hendrik R. A. , Wacker, Anna , Richter, Christian , Comte, Arnaud , Berntenis, Nikolaos , Schmucki, Roland , Hatje, Klas , Petermann, Olivier , Chiriano, Gianpaolo , Perozzo, Remo , Sciarra, Daniel , Konieczny, Piotr , Faustino, Ignacio , Fournet, Guy , Orozco, Modesto , Artero, Ruben , Metzger, Friedrich , Ebeling, Martin , Goekjian, Peter , Joseph, Benoît , Schwalbe, Harald and Scapozza, Leonardo
Publisher
London: Nature Publishing Group UK
Journal title
Language
English
Formats
Publication information
Publisher
London: Nature Publishing Group UK
Subjects
More information
Scope and Contents
Contents
Modification of
SMN2
exon 7 (E7) splicing is a validated therapeutic strategy against spinal muscular atrophy (SMA). However, a target-based approach to identify small-molecule E7 splicing modifiers has not been attempted, which could reveal novel therapies with improved mechanistic insight. Here, we chose as a target the stem-loop RNA struct...
Alternative Titles
Full title
Targeting RNA structure in SMN2 reverses spinal muscular atrophy molecular phenotypes
Authors, Artists and Contributors
Author / Creator
Tessaro, Francesca
Jonker, Hendrik R. A.
Wacker, Anna
Richter, Christian
Comte, Arnaud
Berntenis, Nikolaos
Schmucki, Roland
Hatje, Klas
Petermann, Olivier
Chiriano, Gianpaolo
Perozzo, Remo
Sciarra, Daniel
Konieczny, Piotr
Faustino, Ignacio
Fournet, Guy
Orozco, Modesto
Artero, Ruben
Metzger, Friedrich
Ebeling, Martin
Goekjian, Peter
Joseph, Benoît
Schwalbe, Harald
Scapozza, Leonardo
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_doaj_primary_oai_doaj_org_article_cc1752fb9d394b0fb636e7fca670afcc
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_doaj_primary_oai_doaj_org_article_cc1752fb9d394b0fb636e7fca670afcc
Other Identifiers
ISSN
2041-1723
E-ISSN
2041-1723
DOI
10.1038/s41467-018-04110-1
