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Generation and characterization of human iPSC-derived NPC1 I1061T/I10161T i3Neurons as a model for NPC1 disease.

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Title: Generation and characterization of human iPSC-derived NPC1 I1061T/I10161T i3Neurons as a model for NPC1 disease.
Authors: Salhotra S; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.; Equivalent contributions., Cawley NX; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.; Equivalent contributions., White C; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA., Kang I; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA., Prabhu A; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA., Davidson CD; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA., Wassif CA; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA., Porter FD; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
Source: BioRxiv : the preprint server for biology [bioRxiv] 2026 Feb 13. Date of Electronic Publication: 2026 Feb 13.
Publication Type: Journal Article; Preprint
Journal Info: Country of Publication: United States NLM ID: 101680187 Publication Model: Electronic Cited Medium: Internet ISSN: 2692-8205 (Electronic) Linking ISSN: 26928205 NLM ISO Abbreviation: bioRxiv Subsets: PubMed not MEDLINE
Database: MEDLINE Ultimate
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DbLabel: MEDLINE Ultimate
An: 41726996
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PubType: Academic Journal
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  Data: Generation and characterization of human iPSC-derived NPC1 <superscript>I1061T/I10161T</superscript> i<superscript>3</superscript>Neurons as a model for NPC1 disease.
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  Data: <searchLink fieldCode="AU" term="%22Salhotra+S%22">Salhotra S</searchLink>; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.; Equivalent contributions.<br /><searchLink fieldCode="AU" term="%22Cawley+NX%22">Cawley NX</searchLink>; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.; Equivalent contributions.<br /><searchLink fieldCode="AU" term="%22White+C%22">White C</searchLink>; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Kang+I%22">Kang I</searchLink>; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Prabhu+A%22">Prabhu A</searchLink>; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Davidson+CD%22">Davidson CD</searchLink>; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Wassif+CA%22">Wassif CA</searchLink>; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.<br /><searchLink fieldCode="AU" term="%22Porter+FD%22">Porter FD</searchLink>; Section on Molecular Dysmorphology, Division of Translational Medicine, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, Bethesda, Maryland, USA.
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  Data: <searchLink fieldCode="JN" term="%22101680187%22">BioRxiv : the preprint server for biology</searchLink> [bioRxiv] 2026 Feb 13. <i>Date of Electronic Publication: </i>2026 Feb 13.
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  Data: <i>Country of Publication: </i>United States <i>NLM ID: </i>101680187 <i>Publication Model: </i>Electronic <i>Cited Medium: </i>Internet <i>ISSN: </i>2692-8205 (Electronic) <i>Linking ISSN: </i><searchLink fieldCode="IS" term="%2226928205%22">26928205 </searchLink><i>NLM ISO Abbreviation: </i>bioRxiv <i>Subsets: </i>PubMed not MEDLINE
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        Value: 10.64898/2026.02.11.705111
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        Text: English
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      – TitleFull: Generation and characterization of human iPSC-derived NPC1 I1061T/I10161T i3Neurons as a model for NPC1 disease.
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              Text: 2026 Feb 13
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