Antisense oligonucleotides and spinal muscular atrophy: skipping along.

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).

• Authors: Arthur H M Burghes, Vicki L McGovern
• Journal: Genes & development (Genes Dev) Vol. 24 Issue 15 Pg. 1574-9 (Aug 01 2010) ISSN: 1549-5477 [Electronic] United States
• PMID: 20679391 (Publication Type: Comment, Journal Article, Research Support, N.I.H., Extramural)

Chemical References

• Nerve Tissue Proteins
• Oligonucleotides, Antisense
• Survival of Motor Neuron 2 Protein

Topics

• Alternative Splicing (drug effects, genetics)
• Animals
• Disease Models, Animal
• Humans
• Introns
• Mice
• Muscular Atrophy, Spinal (genetics, metabolism, therapy)
• Nerve Tissue Proteins (genetics, metabolism)
• Oligonucleotides, Antisense (chemistry, genetics, metabolism, pharmacology)
• RNA Splicing (drug effects)
• Survival of Motor Neuron 2 Protein (genetics)