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2.4. AAV Construct

BO Bernie S. Owusu-Yaw
YZ Yongzhi Zhang
LG Lilyan Garrett
AY Alvin Yao
KS Kai Shing
AB Ana Rita Batista
MS Miguel Sena-Esteves
JU Jaymin Upadhyay
KK Kimberly Kegel-Gleason
NT Nick Todd
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The ss-AAV9-U6-miR10150-CBA-GFP virus was kindly donated to us by Dr. Miguel Sena-Esteves and Dr. Ana Rita Batista for use in this study. The vector contains a U6 promoter, which drives the transcription of miR10150 targeting bp10150 in the 3′ untranslated region of the mouse and human HTT gene, and the chimeric cytomegalovirus enhancer/chicken β-actin (CBA) promoter, which drives the expression of GFP (Figure 2). While this study was not designed to assess the efficacy of HTT lowering, the construct containing the micro-RNA, miR10150, was used in this study in order to assess the safety of the full construct.

Diagram of AAV9-U6-miRNA10150-CBA-GFP construct.

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Licensee MDPI, Basel, Switzerland. This article is an open access article distributed under the terms and conditions of the Creative Commons Attribution (CC BY) license (https://creativecommons.org/licenses/by/4.0/).

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