Immune dysfunction in Niemann‐Pick disease type C - Platt - 2016 - Journal of Neurochemistry - Wiley Online Library
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Last updated 25 março 2025


Journal of Cellular Physiology, Cell Biology Journal

A novel strategy for delivering Niemann‐Pick type C2 proteins across the blood–brain barrier using the brain endothelial‐specific AAV‐BR1 virus - Rasmussen - 2023 - Journal of Neurochemistry - Wiley Online Library

Lysosomal storage disorders: pathology within the lysosome and beyond - Kielian - 2019 - Journal of Neurochemistry - Wiley Online Library

A novel strategy for delivering Niemann‐Pick type C2 proteins across the blood–brain barrier using the brain endothelial‐specific AAV‐BR1 virus - Rasmussen - 2023 - Journal of Neurochemistry - Wiley Online Library

Miglustat in Niemann-Pick disease type C patients: a review, Orphanet Journal of Rare Diseases

Immune dysfunction in Niemann‐Pick disease type C - Platt - 2016 - Journal of Neurochemistry - Wiley Online Library

Complex neuroprotective and neurotoxic effects of histone deacetylases - Thomas - 2018 - Journal of Neurochemistry - Wiley Online Library

A novel strategy for delivering Niemann‐Pick type C2 proteins across the blood–brain barrier using the brain endothelial‐specific AAV‐BR1 virus - Rasmussen - 2023 - Journal of Neurochemistry - Wiley Online Library
Intracellular Delivery of β-Galactosidase Enzyme Using Arginase-Responsive Dextran Sulfate/Poly-l-arginine Capsule for Lysosomal Storage Disorder

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice
Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts

10: Types A and B Niemann–Pick Disease

Contribution of ceramides metabolism in psychiatric disorders - Bernal‐Vega - 2023 - Journal of Neurochemistry - Wiley Online Library

Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts
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