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

Hadaczek P, Stanek L, Ciesielska A, Sudhakar V, Samaranch L, Pivirotto P, Bringas J, O’Riordan C, Mastis B, San Sebastian W, Forsayeth J, Cheng SH, Bankiewicz KS, Shihabuddin LS. Mol Ther Methods Clin Dev.

Silencing mutant huntingtin by adeno-associated virus-mediated RNA interference ameliorates disease manifestations in the YAC128 mouse model of Huntington’s disease.

Stanek LM, Sardi SP, Mastis B, Richards AR, Treleaven CM, Taksir T, Misra K, Cheng SH, Shihabuddin LS. Hum Gene Ther. 2014 May;25(5):461-74. (Sanofi-Genzyme)


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