Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapies.

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Bibliographic Details
Title: Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapies.
Authors: Azaria RD; Department of Pathology, University of Michigan Medical School, Ann Arbor, Michigan, USA., Correia AB; Department of Pathology, University of Michigan Medical School, Ann Arbor, Michigan, USA., Schache KJ; Department of Pathology, University of Michigan Medical School, Ann Arbor, Michigan, USA., Zapata M; Department of Chemistry, University of Illinois Chicago, Illinois, USA., Pathmasiri KC; Department of Chemistry, University of Illinois Chicago, Illinois, USA., Mohanty V; Department of Chemistry, University of Illinois Chicago, Illinois, USA., Nannapaneni DT; Department of Chemistry & Biochemistry and., Ashfeld BL; Department of Chemistry & Biochemistry and.; Warren Family Center for Drug Discovery, University of Notre Dame, Notre Dame, Indiana, USA., Helquist P; Department of Chemistry & Biochemistry and., Wiest O; Department of Chemistry & Biochemistry and., Ohgane K; Department of Chemistry, Ochanomizu University, Tokyo, Japan., Li Q; HitGen Inc, Chengdu, China., Fredenburg RA; Ara Parseghian Medical Research Fund at Notre Dame University, Notre Dame, Indiana, USA., Blagg BS; Department of Chemistry & Biochemistry and.; Warren Family Center for Drug Discovery, University of Notre Dame, Notre Dame, Indiana, USA., Cologna SM; Department of Chemistry, University of Illinois Chicago, Illinois, USA., Schultz ML; Stead Family Department of Pediatrics, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA., Lieberman AP; Department of Pathology, University of Michigan Medical School, Ann Arbor, Michigan, USA.
Source: JCI insight [JCI Insight] 2024 Aug 29; Vol. 9 (20). Date of Electronic Publication: 2024 Aug 29.
Publication Type: Journal Article; Research Support, N.I.H., Extramural; Research Support, U.S. Gov't, Non-P.H.S.; Research Support, Non-U.S. Gov't
Journal Info: Publisher: American Society for Clinical Investigation Country of Publication: United States NLM ID: 101676073 Publication Model: Electronic Cited Medium: Internet ISSN: 2379-3708 (Electronic) Linking ISSN: 23793708 NLM ISO Abbreviation: JCI Insight Subsets: MEDLINE
Database: MEDLINE Ultimate
Description
ISSN:2379-3708
DOI:10.1172/jci.insight.179525