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Enrichment of NPC1-deficient cells with the lipid LBPA stimulates autophagy, improves lysosomal function, and reduces cholesterol storage.
Ilnytska O, Lai K, Gorshkov K, Schultz ML, Tran BN, Jeziorek M, Kunkel TJ, Azaria RD, McLoughlin HS, Waghalter M, Xu Y, Schlame M, Altan-Bonnet N, Zheng W, Lieberman AP, Dobrowolski R, Storch J. Ilnytska O, et al. Among authors: schultz ml. J Biol Chem. 2021 Jul;297(1):100813. doi: 10.1016/j.jbc.2021.100813. Epub 2021 May 21. J Biol Chem. 2021. PMID: 34023384 Free PMC article.
Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapies.
Azaria RD, Correia AB, Schache KJ, Zapata M, Pathmasiri KC, Mohanty V, Nannapaneni DT, Ashfeld BL, Helquist P, Wiest O, Ohgane K, Li Q, Fredenburg RA, Blagg BS, Cologna SM, Schultz ML, Lieberman AP. Azaria RD, et al. Among authors: schultz ml. JCI Insight. 2024 Aug 29;9(20):e179525. doi: 10.1172/jci.insight.179525. JCI Insight. 2024. PMID: 39207850 Free PMC article.
51 results