Gene therapy for the treatment of Niemann-Pick disease type C1: Comparison of AAV9 to a novel serotype, AAV-PHP.B von Davidson, Cristin, Gibson, Alana, Gu, Tansy, Chandler, Randy, Deverman, Benjamin, Gradinaru, Viviana, Venditti, Charles, Pavan, William

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Improved disease amelioration with combination therapy for Niemann-Pick disease type C1 von Davidson, Cristin, Gibson, Alana, Gu, Tansy, Ali, Nafeeza, Chandler, Randy, Venditti, Charles, Walkley, Steven U., Pavan, William

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Maternal immune activation modifies the course of Niemann-pick disease, type C1 in a gender specific manner von Cougnoux, Antony, Fellmeth, Mason, Gu, Tansy, Davidson, Cristin D., Gibson, Alana L., Pavan, William J., Porter, Forbes D.

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Improved systemic AAV gene therapy with a neurotrophic capsid in Niemann-Pick disease type C1 mice von Davidson, Cristin D, Gibson, Alana L, Gu, Tansy, Baxter, Laura L, Deverman, Benjamin E, Beadle, Keith, Incao, Arturo A, Rodriguez-Gil, Jorge L, Fujiwara, Hideji, Jiang, Xuntian, Chandler, Randy J, Ory, Daniel S, Gradinaru, Viviana, Venditti, Charles P, Pavan, William J

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