Sage Journals: Discover world-class research

Retricted access

Research article

First published 2026-4

An Analysis of Biomarkers for the Evaluation of Gene Therapy in Niemann–Pick Disease Type C1 Mice

Chika Watanabe, Masamitsu Maekawa, Eriko Jimbo [...]

Human Gene Therapy

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Research article

First published 2025-04

Prevalence of Neutralizing Antibodies to AAV2 and AAV9 in Individuals with Niemann-Pick Disease,Type C1

Avani V. Mylvara, Cristin D. Davidson, Derek Alexander [...]

Human Gene Therapy

Open access

Research article

First published 2021-06

Gene Therapy in a Mouse Model of Niemann–Pick Disease Type C1

Yoshie Kurokawa, Hitoshi Osaka, Takeshi Kouga [...]

Human Gene Therapy

Open access

Research article

First published 2017-06

Analytical Characterization of Methyl-β-Cyclodextrin for Pharmacological Activity to Reduce Lysosomal Cholesterol Accumulation in Niemann-Pick Disease Type C1 Cells

Rong Li, Jon Hao, Hideji Fujiwara [...]

ASSAY and Drug Development Technologies

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Research article

First published 2021-06

Gene Therapy Moves Forward for Niemann–Pick Disease Type C1

R. Scott McIvor

Human Gene Therapy

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Research article

First published 2018-1

Gait,Balance,and Coordination Impairments in Niemann Pick Disease,Type C1

Ashwini Sansare, Cris Zampieri, Katharine Alter [...]

Journal of Child Neurology

Free access

Research article

First published 2011-8

Human Umbilical Cord Blood-Derived Mesenchymal Stem Cells Protect against Neuronal Cell Death and Ameliorate Motor Deficits in Niemann Pick Type C1 Mice

Yoojin Seo, Se-Ran Yang, Min Ki Jee [...]

Cell Transplantation

Open access

Case report

First published 2022

Lamellar Bodies in Podocytes Associated With Compound Heterozygous Mutations for Niemann Pick Type C1 Mimicking Fabry Disease,a Case Report

Pairach Pintavorn, Stephanie Munie, Sweta Munagapati

Canadian Journal of Kidney Health and Disease

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Research article

First published 2013-12

New Niemann-Pick Type C1 Gene Mutation Associated With Very Severe Disease Course and Marked Early Cerebellar Vermis Atrophy

Carlo Fusco, Angelo Russo, Daniela Galla [...]

Journal of Child Neurology

Free access

Research article

First published 2026-1-21

Swallowing assessments: A unique methodology to enhance medical research

Beth I Solomon, Andrea M Muñoz, Ninet Sinaii [...]

Research Methods in Medicine & Health Sciences

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