Antisense oligonucleotides and spinal muscular atrophy: skipping along.
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).
Burghes
Arthur H M
AH
Department of Molecular and Cellular Biochemistry, The Ohio State University, Columbus, Ohio 43210, USA. Burghes.1@osu.edu
McGovern
Vicki L
VL
D017398:Alternative Splicing / Q000187:drug effects / Q000235:genetics; D000818:Animals; D004195:Disease Models, Animal; D006801:Humans; D007438:Introns; D051379:Mice; D009134:Muscular Atrophy, Spinal / Q000235:genetics / Q000378:metabolism* / Q000628:therapy; D009419:Nerve Tissue Proteins / Q000235:genetics / Q000378:metabolism; D016376:Oligonucleotides, Antisense / Q000737:chemistry / Q000235:genetics / Q000378:metabolism* / Q000494:pharmacology; D012326:RNA Splicing / Q000187:drug effects; D055540:Survival of Motor Neuron 2 Protein / Q000235:genetics
D009419:Nerve Tissue Proteins; D016376:Oligonucleotides, Antisense; D055540:Survival of Motor Neuron 2 Protein