ArticleClinical lipidology2010
Niemann-Pick type C pathogenesis and treatment: from statins to sugars.
Article in Clinical lipidology, 2010. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
What it found
Each row is one number read from the abstract, on the scale the paper reported it, with its interval. Left of the dashed line favours the treatment, right favours the comparator. Under each row is the sentence it came from. New to these charts? A ten-minute tutorial.
The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
The trial behind it
Trials whose registry record cites this paper, or whose number appears in the abstract. A trial that started after this paper was published is citing it as background, not reporting it.
Neither the registry nor the abstract names a trial number. If this is a trial report, that itself is worth knowing.
Who cites it
24 citing papers in PubMed, 46 citations in OpenAlex.
- Hyperactivation of Hedgehog signaling impedes myelin development and repair via cholesterol dysregulation in oligodendrocytes.iScience · 2024Article
- PI(4,5)PAdvances in experimental medicine and biology · 2023Article
- Bidirectional Control between Cholesterol Shuttle and Purine Signal at the Central Nervous System.International journal of molecular sciences · 2022Review
- Assessment of Neurodegenerative Changes in Turkeys Fed Diets with Different Proportions of Arginine and Methionine Relative to Lysine.Animals : an open access journal from MDPI · 2022Article
- Neurotrophins as Key Regulators of Cell Metabolism: Implications for Cholesterol Homeostasis.International journal of molecular sciences · 2021Review
- Potential Disease-Modifying Effects of Lithium Carbonate in Niemann-Pick Disease, Type C1.Frontiers in pharmacology · 2021Article
- Human iPSC-Based Models for the Development of Therapeutics Targeting Neurodegenerative Lysosomal Storage Diseases.Frontiers in molecular biosciences · 2020Review
- Cholesterol intake and statin use regulate neuronal G protein-gated inwardly rectifying potassium channels.Journal of lipid research · 2019Article
- Reduction of lipid accumulation rescues Bietti's crystalline dystrophy phenotypes.Proceedings of the National Academy of Sciences of the United States of America · 2018Article
- Potential Link Between Proprotein Convertase Subtilisin/Kexin Type 9 and Alzheimer's Disease.International journal of biomedical investigation · 2018Article
- Pneumococcal Immunization Reduces Neurological and Hepatic Symptoms in a Mouse Model for Niemann-Pick Type C1 Disease.Frontiers in immunology · 2018Article
- Generation of patient specific human neural stem cells from Niemann-Pick disease type C patient-derived fibroblasts.Oncotarget · 2017Article
- Small molecules as therapeutic agents for inborn errors of metabolism.Journal of inherited metabolic disease · 2017Review
- Oleic Acid and Hydroxytyrosol Inhibit Cholesterol and Fatty Acid Synthesis in C6 Glioma Cells.Oxidative medicine and cellular longevity · 2017Article
- Cholesterol metabolism and homeostasis in the brain.Protein & cell · 2015Review
- Recent advances in the diagnosis and treatment of niemann-pick disease type C in children: a guide to early diagnosis for the general pediatrician.International journal of pediatrics · 2015Review
- Role of LDL cholesterol and endolysosomes in amyloidogenesis and Alzheimer's disease.Journal of neurology & neurophysiology · 2014Article
- Niemann-Pick Disease Type C: Induced Pluripotent Stem Cell-Derived Neuronal Cells for Modeling Neural Disease and Evaluating Drug Efficacy.Journal of biomolecular screening · 2014Article
- Lipid metabolism in Alzheimer's disease.Neuroscience bulletin · 2014Review
- Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
2 authors at 1 institution in 1 country.
Funding
Abstract
The isolation of the causative genes for Niemann-Pick type C disease, a panethnic lysosomal lipid storage disorder, has provided models of how sterols and other lipids such as glycosphingolipids traverse the membranes of eukaryotic cells. Unfortunately, these molecular advances have yet to reciprocate with a cure for this devastating neurodegenerative disorder where neuronal replenishment will most likely yield the greatest benefit. In the meantime, stabilizing treatment strategies based on the removal of presumably toxic metabolites are in place. For example, the small molecule inhibition of glucosylceramide synthase by miglustat limits ganglioside accumulation and is now the only approved treatment of Niemann-Pick type C. In addition, 2-hydroxypropyl-B-cyclodextrin, a lipid chelator, relieves the lysosomal to endoplasmic reticulum blockage and markedly increases the life expectancy of the murine model. Ultimately, these strategies, targeting the primary biochemical lesion in these cells, and others will likely be combined to provide a synergistic cocktail approach to treating this disease.
Identifiers
What OpenQuestion holds
Registered trials
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.