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Mutant Induced Neurons and Humanized Mice Enable Identification of NiemannPick Type C1 Proteostatic Therapies

Ruth D. Azaria, Adele B. Correia,Kylie J. Schache, Manuela Zapata,Koralege C. Pathmasiri, Varshasnata Mohanty, Dharma T. Nannapaneni,Brandon L. Ashfeld,Paul Helquist,Olaf Wiest,Kenji Ohgane, Qingqing Li, Ross A. Fredenburg,Brian S. J. Blagg,Stephanie M. Cologna,Mark L. Schultz,Andrew P. Lieberman

JCI INSIGHT(2024)

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