ReviewThe FEBS journal2013
The potential of histone deacetylase inhibitors in Niemann - Pick type C disease.
Review in The FEBS journal, 2013. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 11 papers.
What it found
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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
11 citing papers in PubMed, 19 citations in OpenAlex.
- Epidrug screening identifies type I PRMT inhibitors as modulators of lysosomal exocytosis and drug sensitivity in cancers.Cell death & disease · 2025Article
- Current Challenges in Understanding the Cellular and Molecular Mechanisms in Niemann-Pick Disease Type C1.International journal of molecular sciences · 2019Review
- HDAC inhibitors rescue multiple disease-causing CFTR variants.Human molecular genetics · 2019Article
- Generation of patient specific human neural stem cells from Niemann-Pick disease type C patient-derived fibroblasts.Oncotarget · 2017Article
- FTY720/fingolimod increases NPC1 and NPC2 expression and reduces cholesterol and sphingolipid accumulation in Niemann-Pick type C mutant fibroblasts.FASEB journal : official publication of the Federation of American Societies for Experimental Biology · 2017Article
- Characterization of cholesterol homeostasis in sphingosine-1-phosphate lyase-deficient fibroblasts reveals a Niemann-Pick disease type C-like phenotype with enhanced lysosomal CaScientific reports · 2017Article
- Modeling Niemann Pick type C1 using human embryonic and induced pluripotent stem cells.Brain research · 2017Review
- Neuronal gene repression in Niemann-Pick type C models is mediated by the c-Abl/HDAC2 signaling pathway.Biochimica et biophysica acta · 2016Article
- Therapies for ataxias.Current treatment options in neurology · 2014Article
- Sphingolipids and mitochondrial function, lessons learned from yeast.Microbial cell (Graz, Austria) · 2014Review
- Epigenetic regulation of cholesterol homeostasis.Frontiers in genetics · 2014Review
Corrections and comments
PubMed lists nothing against this paper. Absence here is not a guarantee, only a check that was made.
Authors and funding
3 authors at 1 institution in 1 country.
Funding
Abstract
Niemann-Pick type C (NPC) disease is a fatal complex neurodegenerative lysosomal storage disorder caused by genetic mutations in the proteins NPC1 (95% of patients) or NPC2 that decrease intracellular cholesterol trafficking, resulting in accumulation of unesterified cholesterol and sphingolipids in lysosomal storage organelles. Unfortunately, treatment options for NPC disease are still very limited, although miglustat, which inhibits glucosylceramide synthase, thus limiting ganglioside accumulation, has been approved for treatment of NPC disease. Here we discuss advances in the understanding of NPC1 and its functions, and several new strategies for interfering with cholesterol and sphingolipid accumulation in NPC1-null mice. We also describe several recent studies demonstrating that histone deacetylase inhibitors may correct cholesterol-storage defects in human NPC1 mutant fibroblasts by increasing expression of the low-transport-activity NPC1 mutant protein. These studies may lead to development of new therapeutic approaches for treatment of NPC disease.
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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.