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Comment
. 2012 Jun 21;74(6):964-6.
doi: 10.1016/j.neuron.2012.06.001.

"Huntingtin holiday": progress toward an antisense therapy for Huntington's disease

Xiao-Hong Lu  1 , X William Yang
Affiliations
Comment

"Huntingtin holiday": progress toward an antisense therapy for Huntington's disease

Xiao-Hong Lu et al. Neuron. .

Abstract

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.

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Figures

Figure 1
Figure 1. Transient ASO-Mediated Htt Lowering Produces Sustained Therapeutic Effect in HD Mice
(A) A schematic to show ASOs with phosphorothioate (PS) and 2′-O-methoxyethyl (MOE) modifications that can target Htt RNA to form RNA/DNA duplex and activate RNase H-mediated degradation of Htt mRNA. (B) A schematic to illustrate the “Huntingtin Holiday” effect, a transient lowering of mHtt by ASOs in HD mice results in sustained therapeutic benefit beyond the period of disease protein suppression.
See this image and copyright information in PMC

Comment on

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