Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington’s disease
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...
Main Authors: | , , , , , , , , , , , , , |
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Format: | Online |
Language: | English |
Published: |
Nature Publishing Group
2016
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Online Access: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC4926858/ |