A murine Niemann-Pick C1 I1061T knock-In model recapitulates the pathological features of the most prevalent human disease allele

Maria Praggastis, Brett Tortelli, Jessie Zhang, Hideji Fujiwara, Rohini Sidhu, Anita Chacko, Zhouji Chen, Chan Chung, Andrew P. Lieberman, Jakub Sikora, Cristin Davidson, Steven U. Walkley, Nina H. Pipalia, Frederick R. Maxfield, Jean E. Schaffer, Daniel S. Ory

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Medicine & Life Sciences