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Open Access Publications from the University of California

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

  • Author(s): Bankiewicz, Krzysztof
  • Hadaczek, P
  • Stanek, L
  • Ciesielska, A
  • Sudhakar, V
  • Samaranch, L
  • Pivirotto, P
  • Bringas, J
  • O'Riordan, C
  • Mastis, B
  • San, W
  • et al.

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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

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