Discovery and Optimization of Small Molecule Splicing Modifiers of Survival Motor Neuron 2 as a Treatment for Spinal Muscular Atrophy
Author:
Affiliation:
1. PTC Therapeutics, Inc., 100 Corporate Court, South Plainfield, New Jersey 07080, United States
Funder
Spinal Muscular Atrophy Foundation
Publisher
American Chemical Society (ACS)
Subject
Drug Discovery,Molecular Medicine
Link
https://pubs.acs.org/doi/pdf/10.1021/acs.jmedchem.6b00460
Reference27 articles.
1. Identification and characterization of a spinal muscular atrophy-determining gene
2. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy
3. A degron created by SMN2 exon 7 skipping is a principal contributor to spinal muscular atrophy severity
4. Regulation of SMN Protein Stability
5. Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice
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