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Open access

Article commentary

First published 2024

Unraveling the Connection: Cholesterol,Calcium Signaling,and Neurodegeneration

Maria Casas, Eamonn J Dickson

Neuroscience Insights

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

Open access

Article commentary

First published 2019

Cholesterol Overload: Contact Sites to the Rescue!

Carlos Enrich, Carles Rentero, Thomas Grewal [...]

Contact: The Journal of Inter-Organelle Communication

Open access

Brief report

First published 2015-08

Cyclodextrin Alters GABAergic Input to CA1 Pyramidal Cells in Wild-Type But Not in NPC1-Deficient Mice

Moritz J. Frech, Michael Rabenstein, Katja Bovensiepen [...]

BioResearch Open Access

Retricted access

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

Retricted access

Research article

First published 2012-6-18

Niemann-Pick C1 Mice,a Model of “Juvenile Alzheimer's Disease”,with Normal Gene Expression in Neurons and Fibrillary Astrocytes Show Long Term Survival and Delayed Neurodegeneration

Ivan Borbon, John Totenhagen, Maria Teresa Fiorenza [...]

Journal of Alzheimer’s Disease

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

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

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

Open access

Review article

First published 2024

Channels,Transporters,and Receptors at Membrane Contact Sites

Maria Casas, Eamonn James Dickson

Contact: The Journal of Inter-Organelle Communication

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