widespread aav1- and aav2-mediated transgene expression in the nonhuman primate brain: implications for huntington's disease

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ID: 187932
2016
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Abstract
Huntington's disease (HD) is caused by a toxic gain-of-function associated with the expression of the mutant huntingtin (htt) protein. Therefore, the use of RNA interference to inhibit Htt expression could represent a disease-modifying therapy. The potential of two recombinant adeno-associated viral vectors (AAV), AAV1 and AAV2, to transduce the cortico-striatal tissues that are predominantly affected in HD was explored. Green fluorescent protein was used as a reporter in each vector to show that both serotypes were broadly distributed in medium spiny neurons in the striatum and cortico-striatal neurons after infusion into the putamen and caudate nucleus of nonhuman primates (NHP), with AAV1-directed expression being slightly more robust than AAV2-driven expression. This study suggests that both serotypes are capable of targeting neurons that degenerate in HD, and it sets the stage for the advanced preclinical evaluation of an RNAi-based therapy for this disease.
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hadaczek2016molecularwidespread Use this key to autocite in the manuscript while using SciMatic Manuscript Manager or Thesis Manager
Authors Piotr Hadaczek, Lisa Stanek, Agnieszka Ciesielska, Vivek Sudhakar, Lluis Samaranch, Philip Pivirotto, John Bringas, Catherine O'Riordan, Bryan Mastis, Waldy San Sebastian, John Forsayeth, Seng H Cheng, Krystof S Bankiewicz, Lamya S Shihabuddin
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Journal international journal of zoology
Year 2016
DOI
10.1038/mtm.2016.37
URL
http://www.sciencedirect.com/science/article/pii/S2329050117300116
Keywords
geneticscytology

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