Journal
GENES & DEVELOPMENT
Volume 24, Issue 15, Pages 1574-1579Publisher
COLD SPRING HARBOR LAB PRESS, PUBLICATIONS DEPT
DOI: 10.1101/gad.1961710
Keywords
Spinal muscular atrophy; SMN2; antisense oligonucleotide; splicing correction; spinal cord; mouse models
Categories
Funding
- NIH [NS038650, HD060586, RC2 NS069476]
- MDA
- Families of SMA
- Madison, Matthew, Preston, and Cade and Katelyn funds
- SMA Angels (Savannah)
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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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