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Genes Dev. 2010 Aug 1;24(15):1574-9. doi: 10.1101/gad.1961710.

Antisense oligonucleotides and spinal muscular atrophy: skipping along.

Author information

1
Department of Molecular and Cellular Biochemistry, The Ohio State University, Columbus, Ohio 43210, USA. Burghes.1@osu.edu

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).

PMID:
20679391
PMCID:
PMC2912553
DOI:
10.1101/gad.1961710
[Indexed for MEDLINE]
Free PMC Article
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