ISS-N1 makes the first FDA-approved drug for spinal muscular atrophy
Author:
Abstract
Publisher
Walter de Gruyter GmbH
Subject
General Neuroscience
Link
https://www.degruyter.com/document/doi/10.1515/tnsci-2017-0001/pdf
Reference71 articles.
1. A short antisense oligonucleotide masking a unique intronic motif prevents skipping of a critical exon in spinal muscular atrophy;RNA Biol,2009
2. Disruption of an SF2/ASF-dependent exonic splicing enhancer in SMN2 causes spinal muscular atrophy in the absence of SMN1;Nat. Genet,2002
3. Oxidative Stress Triggers Body-Wide Skipping of Multiple Exons of the Spinal Muscular Atrophy Gene;Plos One,2016
4. Identification of a cis-acting element for the regulation of SMN exon 7 splicing;J. Biol. Chem,2002
5. Bifunctional RNAs Targeting the Intronic Splicing Silencer N1 Increase SMN Levels and Reduce Disease Severity in an Animal Model of Spinal Muscular Atrophy;Mol. Ther,2012
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