Xiao-Hong Lu, X. William Yang Neuron

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“Huntingtin Holiday”: Progress toward an Antisense Therapy for Huntington's Disease Xiao-Hong Lu, X. William Yang Neuron Volume 74, Issue 6, Pages 964-966 (June 2012) DOI: 10.1016/j.neuron.2012.06.001 Copyright © 2012 Elsevier Inc. Terms and Conditions

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 2012 74, 964-966DOI: (10.1016/j.neuron.2012.06.001) Copyright © 2012 Elsevier Inc. Terms and Conditions