Big win possible for Ionis/Biogen antisense drug in muscular atrophy
Author:
Publisher
Springer Science and Business Media LLC
Subject
Biomedical Engineering,Molecular Medicine,Applied Microbiology and Biotechnology,Bioengineering,Biotechnology
Link
http://www.nature.com/articles/nbt1016-1002.pdf
Reference8 articles.
1. Identification and characterization of a spinal muscular atrophy-determining gene
2. Results from a phase 1 study of nusinersen (ISIS-SMNRx) in children with spinal muscular atrophy
3. A single nucleotide in the SMN gene regulates splicing and is responsible for spinal muscular atrophy
4. Correction of disease-associated exon skipping by synthetic exon-specific activators
5. Antisense Masking of an hnRNP A1/A2 Intronic Splicing Silencer Corrects SMN2 Splicing in Transgenic Mice
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