Depletion of AADC activity in caudate nucleus and putamen of Parkinson's disease patients; implications for ongoing AAV2-AADC gene therapy trial

Autor: Bankiewicz, Krzysztof, Ciesielska, A, Samaranch, L, San, W, Dickson, DW, Goldman, S, Forsayeth, J, Bankiewicz, KS

Publikováno v: Bankiewicz, Krzysztof; Ciesielska, A; Samaranch, L; San, W; Dickson, DW; Goldman, S; et al.(2017). Depletion of AADC activity in caudate nucleus and putamen of Parkinson's disease patients; implications for ongoing AAV2-AADC gene therapy trial. UC San Francisco: Retrieved from: http://www.escholarship.org/uc/item/13j4600p

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od_______325::3b67322d200f53412a74583dab88fbe3
http://www.escholarship.org/uc/item/13j4600p

Axonal transport of adeno-associated viral vectors is serotype-dependent.

Autor: Salegio, E A¹, Samaranch, L¹, Kells, A P¹, Mittermeyer, G¹, San Sebastian, W¹, Zhou, S², Beyer, J¹, Forsayeth, J¹, Bankiewicz, K S¹

Publikováno v: Gene Therapy. Mar2013, Vol. 20 Issue 3, p348-352. 5p. 1 Black and White Photograph, 5 Diagrams.

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

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

Publikováno v: Bankiewicz, Krzysztof; Hadaczek, P; Stanek, L; Ciesielska, A; Sudhakar, V; Samaranch, L; et al.(2016). Widespread AAV1-and AAV2-mediated transgene expression in the nonhuman primate brain: implications for Huntington's disease.. UC San Francisco: Retrieved from: http://www.escholarship.org/uc/item/9z77c5t3

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 p

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http://www.escholarship.org/uc/item/9z77c5t3

Distribution of nanoparticles throughout the cerebral cortex of rodents and non-human primates: Implications for gene and drug therapy

Autor: Bankiewicz, Krzysztof, Salegio, EA, Streeter, H, Dube, N, Hadaczek, P, Samaranch, L, Kells, AP, Sebastian, WS, Zhai, Y, Bringas, J, Xu, T

Publikováno v: Bankiewicz, Krzysztof; Salegio, EA; Streeter, H; Dube, N; Hadaczek, P; Samaranch, L; et al.(2014). Distribution of nanoparticles throughout the cerebral cortex of rodents and non-human primates: Implications for gene and drug therapy. UC San Francisco: Retrieved from: http://www.escholarship.org/uc/item/775695vz

When nanoparticles/proteins are infused into the brain, they are often transported to distal sites in a manner that is dependent both on the characteristics of the infusate and the region targeted. We have previously shown that adeno-associated virus

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od_______325::1eb018defc1f62abd158aa55b6683afe
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Axonal transport of adeno-associated viral vectors is serotype-dependent

Autor: Bankiewicz, Krzysztof, Salegio, EA, Samaranch, L, Kells, AP, Mittermeyer, G, San, W, Zhou, S, Beyer, J, Forsayeth, J, Bankiewicz, KS

Publikováno v: Bankiewicz, Krzysztof; Salegio, EA; Samaranch, L; Kells, AP; Mittermeyer, G; San, W; et al.(2013). Axonal transport of adeno-associated viral vectors is serotype-dependent. UC San Francisco: Retrieved from: http://www.escholarship.org/uc/item/80c7v92g

We have previously shown that adeno-associated virus type 2 (AAV2) undergoes anterograde axonal transport in rat and non-human primate brain. We screened other AAV serotypes for axonal transport and found that AAV6 is transported almost exclusively i

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=od_______325::3791484266b10fdfd47b00b22e32f952
http://www.escholarship.org/uc/item/80c7v92g