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

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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
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  Data: Mutant induced neurons and humanized mice enable identification of Niemann-Pick type C1 proteostatic therapies.
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  Data: <searchLink fieldCode="AU" term="%22Azaria+RD%22">Azaria RD</searchLink>; Department of Pathology, University of Michigan Medical School, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Correia+AB%22">Correia AB</searchLink>; Department of Pathology, University of Michigan Medical School, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Schache+KJ%22">Schache KJ</searchLink>; Department of Pathology, University of Michigan Medical School, Ann Arbor, Michigan, USA.<br /><searchLink fieldCode="AU" term="%22Zapata+M%22">Zapata M</searchLink>; Department of Chemistry, University of Illinois Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Pathmasiri+KC%22">Pathmasiri KC</searchLink>; Department of Chemistry, University of Illinois Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Mohanty+V%22">Mohanty V</searchLink>; Department of Chemistry, University of Illinois Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Nannapaneni+DT%22">Nannapaneni DT</searchLink>; Department of Chemistry & Biochemistry and.<br /><searchLink fieldCode="AU" term="%22Ashfeld+BL%22">Ashfeld BL</searchLink>; Department of Chemistry & Biochemistry and.; Warren Family Center for Drug Discovery, University of Notre Dame, Notre Dame, Indiana, USA.<br /><searchLink fieldCode="AU" term="%22Helquist+P%22">Helquist P</searchLink>; Department of Chemistry & Biochemistry and.<br /><searchLink fieldCode="AU" term="%22Wiest+O%22">Wiest O</searchLink>; Department of Chemistry & Biochemistry and.<br /><searchLink fieldCode="AU" term="%22Ohgane+K%22">Ohgane K</searchLink>; Department of Chemistry, Ochanomizu University, Tokyo, Japan.<br /><searchLink fieldCode="AU" term="%22Li+Q%22">Li Q</searchLink>; HitGen Inc, Chengdu, China.<br /><searchLink fieldCode="AU" term="%22Fredenburg+RA%22">Fredenburg RA</searchLink>; Ara Parseghian Medical Research Fund at Notre Dame University, Notre Dame, Indiana, USA.<br /><searchLink fieldCode="AU" term="%22Blagg+BS%22">Blagg BS</searchLink>; Department of Chemistry & Biochemistry and.; Warren Family Center for Drug Discovery, University of Notre Dame, Notre Dame, Indiana, USA.<br /><searchLink fieldCode="AU" term="%22Cologna+SM%22">Cologna SM</searchLink>; Department of Chemistry, University of Illinois Chicago, Illinois, USA.<br /><searchLink fieldCode="AU" term="%22Schultz+ML%22">Schultz ML</searchLink>; Stead Family Department of Pediatrics, Carver College of Medicine, University of Iowa, Iowa City, Iowa, USA.<br /><searchLink fieldCode="AU" term="%22Lieberman+AP%22">Lieberman AP</searchLink>; Department of Pathology, University of Michigan Medical School, Ann Arbor, Michigan, USA.
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  Data: <searchLink fieldCode="JN" term="%22101676073%22">JCI insight</searchLink> [JCI Insight] 2024 Aug 29; Vol. 9 (20). <i>Date of Electronic Publication: </i>2024 Aug 29.
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