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

被引：42

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 [神经生物学];

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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