ArticleClinical and experimental pharmacology & physiology2014
Systemic administration of 2-hydroxypropyl-β-cyclodextrin to symptomatic Npc1-deficient mice slows cholesterol sequestration in the major organs and improves liver function.
Article in Clinical and experimental pharmacology & physiology, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 23 papers.
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Who cites it
23 citing papers in PubMed, 37 citations in OpenAlex.
- Therapeutic targeting of local metabolic dysfunction in secondary lymphedema: mechanisms and opportunities.Frontiers in physiology · 2026Review
- Cholesterol crystals in the pathogenesis of atherosclerosis.Nature reviews. Cardiology · 2025Review
- Advances in research on potential therapeutic approaches for Niemann-Pick C1 disease.Frontiers in pharmacology · 2024Review
- Organ Weights inInternational journal of molecular sciences · 2022Article
- Association between serum sphingolipids and necroinflammation of liver tissue pathology in chronic hepatitis B.International journal of medical sciences · 2022Article
- Inhibition of Histone Deacetylases 1, 2, and 3 Enhances Clearance of Cholesterol Accumulation in Niemann-Pick C1 Fibroblasts.ACS pharmacology & translational science · 2021Article
- Application of a glycinated bile acid biomarker for diagnosis and assessment of response to treatment in Niemann-pick disease type C1.Molecular genetics and metabolism · 2020Article
- Delineation of metabolic responses of Npc1Steroids · 2020Article
- Characterization of the Subventricular-Thalamo-Cortical Circuit in the NP-C Mouse Brain, and New Insights Regarding Treatment.Molecular therapy : the journal of the American Society of Gene Therapy · 2019Article
- Review
- Regulation of lipid rafts, angiogenesis and inflammation by AIBP.Current opinion in lipidology · 2019Review
- Synthesis of the Anionic Hydroxypropyl-β-cyclodextrin:Poly(decamethylenephosphate) Polyrotaxane and Evaluation of its Cholesterol Efflux Potential in Niemann-Pick C1 Cells.Journal of materials chemistry. B · 2019Article
- Vascular endothelial growth factor improves the therapeutic effects of cyclodextrin in Niemann-Pick type C mice.Animal cells and systems · 2019Article
- Niemann-Pick C1-deficient mice lacking sterol O-acyltransferase 2 have less hepatic cholesterol entrapment and improved liver function.American journal of physiology. Gastrointestinal and liver physiology · 2018Article
- Targeting toll-like receptor-4 (TLR4)-an emerging therapeutic target for persistent pain states.Pain · 2018Review
- Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder.Scientific reports · 2018Article
- The role of Purkinje cell-derived VEGF in cerebellar astrogliosis in Niemann-Pick type C mice.BMB reports · 2018Article
- 2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1.Current pharmaceutical design · 2017Review
- The Potential of Cyclodextrins as Novel Active Pharmaceutical Ingredients: A Short Overview.Molecules (Basel, Switzerland) · 2016Review
- Pharmacologic Treatment Assigned for Niemann Pick Type C1 Disease Partly Changes Behavioral Traits in Wild-Type Mice.International journal of molecular sciences · 2016Article
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Authors and funding
6 authors at 1 institution in 1 country.
Funding
Abstract
In Niemann-Pick type C (NPC) disease, loss-of-function mutations in either NPC1 or NPC2 result in progressive accumulation of unesterified cholesterol (UC) and glycosphingolipids in all organs, leading to neurodegeneration, pulmonary dysfunction and sometimes liver failure. There is no cure for this disorder. Studies using primarily NPC mouse models have shown that systemic administration of 2-hydroxypropyl-β-cyclodextrin (2HPβCD), starting in early neonatal life, diminishes UC accumulation in most organs, slows disease progression and extends lifespan. The key question now is whether delaying the start of 2HPβCD treatment until early adulthood, when the amount of entrapped UC throughout the body is markedly elevated, has any of the benefits found when treatment begins at 7 days of age. In the present study, Npc1(-/-) and Npc1(+/+) mice were given saline or 2HPβCD subcutaneously at 49, 56, 63 and 70 days of age, with measurements of organ weights, liver function tests and tissue cholesterol levels performed at 77 days. In Npc1(-/-) mice, treatment with 2HPβCD from 49 days reduced whole-liver cholesterol content at 77 days from 33.0 ± 1.0 to 9.1 ± 0.5 mg/organ. Comparable improvements were seen in other organs, such as the spleen, and in the animal as a whole. There was a transient increase in biliary cholesterol concentration in Npc1(-/-) mice after 2HPβCD. Plasma alanine aminotransferase and aspartate aminotransferase activities in 77-day-old 2HPβCD-treated Npc1(-/-) mice were reduced compared with saline-treated controls. The lifespan of Npc1(-/-) mice given 2HPβCD marginally exceeded that of the saline-treated controls (99 ± 1.1 vs 94 ± 1.4 days, respectively; P < 0.05). Thus, 2HPβCD is effective in mobilizing entrapped cholesterol in late-stage NPC disease leading to improved liver function.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.