"Huntingtin Holiday": Progress toward an Antisense Therapy for Huntington's Disease

被引：41
作者：
Lu, Xiao-Hong [1 ,2 ,3 ]
Yang, X. William [1 ,2 ,3 ]
机构：
[1] Univ Calif Los Angeles, David Geffen Sch Med, Ctr Neurobehav Genet, Semel Inst Neurosci & Human Behav, Los Angeles, CA 90095 USA
[2] Univ Calif Los Angeles, David Geffen Sch Med, Dept Psychiat & Biobehav Sci, Los Angeles, CA 90095 USA
[3] Univ Calif Los Angeles, David Geffen Sch Med, Inst Brain Res, Los Angeles, CA 90095 USA
来源：
NEURON | 2012年 / 74卷 / 06期
关键词：
MUTANT HUNTINGTIN; MOUSE MODEL; MICE; OLIGONUCLEOTIDES; NEUROPATHOLOGY; PATHOGENESIS; BRAIN; MOTOR;
D O I：
10.1016/j.neuron.2012.06.001
中图分类号：
Q189 [神经科学];
学科分类号：
071006 ;
摘要：
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.
引用
收藏
页码：964 / 966
页数：3
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