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Scholarly Works (1 results)

Article
Peer Reviewed

Widespread AAV1- and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease.

Bankiewicz, Krzysztof;
Hadaczek, P;
Stanek, L;
Ciesielska, A;
Sudhakar, V;
Samaranch, L;
Pivirotto, P;
Bringas, J;
O'Riordan, C;
Mastis, B;
San, W

UC San Francisco Previously Published Works (2016)

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 potenti