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Xiao-Hong Lu, X. William Yang  Neuron 

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1 “Huntingtin Holiday”: Progress toward an Antisense Therapy for Huntington's Disease 
Xiao-Hong Lu, X. William Yang  Neuron  Volume 74, Issue 6, Pages (June 2012) DOI: /j.neuron Copyright © 2012 Elsevier Inc. Terms and Conditions

2 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. Neuron  , DOI: ( /j.neuron ) Copyright © 2012 Elsevier Inc. Terms and Conditions

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