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

Xiao-Hong Lu; X William Yang

doi:10.1016/j.neuron.2012.06.001

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

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

Authors

Xiao-Hong Lu¹, X William Yang

Affiliation

¹ 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.

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.

Publication types

Comment

MeSH terms

Animals
Huntingtin Protein
Huntington Disease / therapy*
Nerve Tissue Proteins / antagonists & inhibitors*
Nuclear Proteins / antagonists & inhibitors*
Oligodeoxyribonucleotides, Antisense / therapeutic use*

Substances

Htt protein, mouse
Huntingtin Protein
Nerve Tissue Proteins
Nuclear Proteins
Oligodeoxyribonucleotides, Antisense

Abstract

Publication types

MeSH terms

Substances

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