Antisense oligonucleotides and spinal muscular atrophy: skipping along
- 1Department of Molecular and Cellular Biochemistry, The Ohio State University, Columbus, Ohio 43210, USA;
- 2Department of Neurology, The Ohio State University, Columbus, Ohio 43210, USA
Abstract
Antisense oligonucleotides (ASOs) can be used to alter the splicing of a gene and either restore production of a required protein or eliminate a toxic product. In this issue of Genes & Development, Hua and colleagues (pp. 1634–1644) show that ASOs directed against an intron splice silencer (ISS) in the survival motor neuron 2 (SMN2) gene alter the amount of full-length SMN transcript in the nervous system, restoring SMN to levels that could correct spinal muscular atrophy (SMA).
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↵3 Corresponding author.
E-MAIL Burghes.1{at}osu.edu; FAX (614) 292-4118.
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Article is online at http://www.genesdev.org/cgi/doi/10.1101/gad.1961710.
- Copyright © 2010 by Cold Spring Harbor Laboratory Press
