ArticleScientific reports2014
Lysosomal-specific cholesterol reduction by biocleavable polyrotaxanes for ameliorating Niemann-Pick type C disease.
Article in Scientific reports, 2014. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 22 papers.
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Who cites it
22 citing papers in PubMed, 70 citations in OpenAlex.
- APOL4-mediated intracellular cholesterol trafficking is essential for glioblastoma cell growth.BMC cancer · 2025Article
- Lysosomal cholesterol overload in macrophages promotes liver fibrosis in a mouse model of NASH.The Journal of experimental medicine · 2023Article
- Polymer-based drug delivery systems under investigation for enzyme replacement and other therapies of lysosomal storage disorders.Advanced drug delivery reviews · 2023Review
- Supermolecule-Drug Conjugates Based on Acid-Degradable Polyrotaxanes for pH-Dependent Intracellular Release of Doxorubicin.Molecules (Basel, Switzerland) · 2023Article
- Activated cholesterol metabolism is integral for innate macrophage responses by amplifying Myd88 signaling.JCI insight · 2022Article
- Article
- Weakly acidic carboxy group-grafted β-cyclodextrin-threaded acid-degradable polyrotaxanes for modulating protein interaction and cellular internalization.Science and technology of advanced materials · 2021Article
- 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
- Mannosylated Polyrotaxanes for Increasing Cellular Uptake Efficiency in Macrophages through Receptor-Mediated Endocytosis.Molecules (Basel, Switzerland) · 2019Article
- The susceptibility of cochlear outer hair cells to cyclodextrin is not related to their electromotile activity.Acta neuropathologica communications · 2018Article
- Linear Cyclodextrin Polymer Prodrugs as Novel Therapeutics for Niemann-Pick Type C1 Disorder.Scientific reports · 2018Article
- Polysaccharide-Based Controlled Release Systems for Therapeutics Delivery and Tissue Engineering: From Bench to Bedside.Advanced science (Weinheim, Baden-Wurttemberg, Germany) · 2018Review
- Development of Dipicolylamine-Modified Cyclodextrins for the Design of Selective Guest-Responsive Receptors for ATP.Molecules (Basel, Switzerland) · 2018Article
- Crystal structure of the inclusion complex of cholesterol in β-cyclodextrin and molecular dynamics studies.Beilstein journal of organic chemistry · 2018Article
- Hepatocyte ABCA1 Deletion Impairs Liver Insulin Signaling and Lipogenesis.Cell reports · 2017Article
- Pluronic based β-cyclodextrin polyrotaxanes for treatment of Niemann-Pick Type C disease.Scientific reports · 2017Article
- PEG-lipid micelles enable cholesterol efflux in Niemann-Pick Type C1 disease-based lysosomal storage disorder.Scientific reports · 2016Article
- Susceptibility of outer hair cells to cholesterol chelator 2-hydroxypropyl-β-cyclodextrine is prestin-dependent.Scientific reports · 2016Article
- Versatile synthesis of end-reactive polyrotaxanes applicable to fabrication of supramolecular biomaterials.Beilstein journal of organic chemistry · 2016Article
- Lysosomal pH-inducible supramolecular dissociation of polyrotaxanes possessing acid-labileScience and technology of advanced materials · 2016Article
Corrections and comments
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Authors and funding
2 authors at 1 institution in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
Niemann-Pick type C (NPC) disease is an autosomal recessive lysosomal trafficking disorder, in which the cholesterols are abnormally accumulated in lysosomes. Recently, the β-cyclodextrin (CD) derivatives are revealed to show therapeutic effect for NPC disease through the removal of accumulated cholesterols in lysosomes. Herein, to enhance the therapeutic effect and reduce the toxicity of β-CD derivatives, biocleavable Pluronic/β-CD-based polyrotaxanes (PRXs) bearing terminal disulfide linkages that can release threaded β-CDs in lysosomes were developed. The biocleavable PRXs show negligible interaction with the plasma membrane, leading to avoiding the toxicity of β-CDs derived from their hydrophobic cavity. Additionally, lysosomal release of threaded β-CDs from biocleavable PRXs by the intracellular cleavage of terminal disulfide linkages is found to achieve approximately 100-fold higher cholesterol removal ability from NPC disease-derived cells than β-CD derivatives. Consequently, the biocleavable PRXs is considered to be a noninvasive and effective therapeutics for NPC disease.
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