Jiang, Xuntian

Lysosomal cholesterol activates mTORC1 via an SLC38A9–Niemann-Pick C1 signaling complex

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AAV gene therapy for Tay-Sachs disease

Flotte, T. R., Cataltepe, O., Puri, A., Batista, A. R., Moser, R., McKenna-Yasek, D., Douthwright, C., Gernoux, G., Blackwood, M., Mueller, C., et al. (2022) · Nature Medicine

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A novel gene editing system to treat both Tay–Sachs and sandhoff diseases

Ou, L., Przybilla, M. J., Tăbăran, A.-F., Overn, P., O’Sullivan, M. G., Jiang, X., Sidhu, R., Kell, P. J., Ory, D. S., & Whitley, C. B. (2020) · Gene Therapy

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