"Huntingtin holiday": progress toward an antisense therapy for Huntington's disease.
Lowering mutant Huntingtin is a consensus therapeutic strategy for Huntington's disease. In this issue of Neuron, Kordasiewicz et al. (2012) show the benefit of transient antisense oligonucleotide (ASO) therapy to degrade Huntingtin mRNA and elicit sustained therapeutic benefit in HD mice.
Semel Institute for Neuroscience and Human Behavior, Department of Psychiatry and Biobehavioral Sciences, Brain Research Institute, David Geffen School of Medicine at University of California, Los Angeles, Los Angeles, CA 90095, USA.
Xiao-Hong
XH
Lu
X William
XW
Yang
D000818:Animals; D000071058:Huntingtin Protein; D006816:Huntington Disease / Q000628:therapy*; D009419:Nerve Tissue Proteins / Q000037:antagonists & inhibitors*; D009687:Nuclear Proteins / Q000037:antagonists & inhibitors*; D020319:Oligodeoxyribonucleotides, Antisense / Q000627:therapeutic use*
C506812:Htt protein, mouse; D000071058:Huntingtin Protein; D009419:Nerve Tissue Proteins; D009687:Nuclear Proteins; D020319:Oligodeoxyribonucleotides, Antisense