Efficacy of a Bicistronic Vector for Correction of Sandhoff Disease in a Mouse Model

por Woodley, Evan, Osmon, Karlaina J.L., Thompson, Patrick, Richmond, Christopher, Chen, Zhilin, Gray, Steven J., Walia, Jagdeep S.
Publicado 2018

Investigating Immune Responses to the scAAV9-HEXM Gene Therapy Treatment in Tay–Sachs Disease and Sandhoff Disease Mouse Models

por Kot, Shalini, Karumuthil-Melethil, Subha, Woodley, Evan, Zaric, Violeta, Thompson, Patrick, Chen, Zhilin, Lykken, Erik, Keimel, John G., Kaemmerer, William F., Gray, Steven J., Walia, Jagdeep S.
Publicado 2021