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Transcriptome of 2-hydroxypropyl-beta-cyclodextrin treatment in Niemann-Pick disease type C1

ABSTRACT: Transcriptome of 2-hydroxypropyl-beta-cyclodextrin treatment in Niemann-Pick disease type C1

PROVIDER: PRJNA717337 | ENA |

REPOSITORIES: ENA

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Transcriptome of 2-hydroxypropyl-beta-cyclodextrin treatment in Niemann-Pick disease type C1

Project description:Transcriptome of 2-hydroxypropyl-beta-cyclodextrin treatment in Niemann-Pick disease type C1

| PRJNA717336 | ENA

Decreased Phosphoinositides Provide Insight to the Neurogenerative Disorder, Niemann-Pick Disease Type C1

Project description:Decreased Phosphoinositides Provide Insight to the Neurogenerative Disorder, Niemann-Pick Disease Type C1

2018-12-28 | MSV000083287 | MassIVE

Hydroxypropyl-β-cyclodextrin spikes local inflammation that induces Th2 and Tfh responses to the coadministered antigen

Project description:Gene expression data from mouse organs after hydroxypropyl-β-cyclodextrin injection Cyclodextrins are commonly used as a safe excipient to enhance the solubility and bioavailability of hydrophobic pharmaceutical agents. In this study, we reprofiled hydroxypropyl-β-cyclodextrin (HP-β-CD) as a vaccine adjuvant, and found that it acts as a potent and unique adjuvant.

2015-02-15 | GSE63332 | GEO

Label-Free Proteomics in the Symptomatic Niemann-Pick, Type C1 Mouse Model

Project description:Quantitative, Label-Free Proteomics in the Symptomatic Niemann-Pick, Type C1 Mouse Model Using Standard Flow Liquid Chromatography and Thermal Focusing Electrospray Ionization

2018-12-03 | MSV000083179 | MassIVE

GNPS - Decreased Phosphoinositides Provide Insight to the Neurogenerative Disorder, Niemann-Pick Disease Type C1

Project description:Decreased Phosphoinositides Provide Insight to the Neurogenerative Disorder, Niemann-Pick Disease Type C1

2018-12-28 | MSV000083287 | GNPS

Niemann-Pick type C fibroblast analysis

Project description:Primary skin fibroblasts from four Niemann-Pick type C patients homozygous for the I1061T mutation and four control individuals were cultured under identical conditions in DMEM containing 10% fetal bovine serum. Cells were harvested at 50-70% confluency. mRNA was isolated with the FastTrack 2.0 mRNA isolation kit according to the manufacturer's instructions (Invitrogen, Carlsbad, CA). A reference RNA comprised of 10 cell lines was used as the control for each hybridized sample (Stratagene, La Jolla, CA). Both sample and reference RNAs were amplified using the MessageAmp II aRNA amplification kit (Ambion, Austin, TX). Set of arrays that are part of repeated experiments Biological Replicate Complex

2007-08-15 | E-GEOD-8770 | biostudies-arrayexpress

Long-Term Treatment of Niemann-Pick Type C1 Disease With Intrathecal 2-Hydroxypropyl-?-Cyclodextrin.

Project description:BACKGROUND:Intrathecal 2-hydoxypropyl-?-cyclodextrin has been found to mobilize cholesterol, extend life, reduce cerebellar pathology, and delay onset of ataxia in the mouse and cat models of Niemann-Pick disease, type C1, a clinically variable progressive and ultimately fatal neurodegenerative storage disorder characterized by endolysosomal accumulation of unesterified cholesterol. OBJECTIVE:In this study, the long-term effects of intrathecal 2-hydoxypropyl-?-cyclodextrin treatment for 2.5 to three years in humans with Niemann-Pick disease, type C, were evaluated. METHODS:Three patients with Niemann-Pick disease, type C, in different stages of progression and displaying varying disease manifestations were treated with intrathecal 2-hydoxypropyl-?-cyclodextrin (VTS-270) delivered by lumbar puncture infusion through an intermediate-size patient population investigational new drug application for expanded access. Disease progression was monitored with the Niemann-Pick disease, type C, Neurological Severity Scale and numerous objective measures of function in five neurological domains typically impacted by the disease: cognitive/language, gait/balance, fine motor, swallowing, and eye movement. RESULTS:No worsening in any domain except eye movements (vertical pursuit gain) was seen for any of the three patients, and in the other domains, improved scores on measures were seen over time for one or more patients. The Niemann-Pick disease type C (NPC) Neurological Severity Scale (NSS) showed stable to slightly improved ratings. CONCLUSIONS:These trajectories are not consistent with the typical trajectory of the disease and suggest that intrathecal 2-hydoxypropyl-?-cyclodextrin has stabilized the disease over an extended period of time, supporting the current phase 2/3 controlled registration trial with VTS-270.

| S-EPMC5857219 | biostudies-literature

Hydroxypropyl-Î²-cyclodextrin spikes local inflammation that induces Th2 and Tfh responses to the coadministered antigen

Project description:Gene expression data from mouse organs after hydroxypropyl-Î²-cyclodextrin injection Cyclodextrins are commonly used as a safe excipient to enhance the solubility and bioavailability of hydrophobic pharmaceutical agents. In this study, we reprofiled hydroxypropyl-Î²-cyclodextrin (HP-Î²-CD) as a vaccine adjuvant, and found that it acts as a potent and unique adjuvant. C57BL/6J mice (6 week old, male, n = 3) were injected i.d. or i.p. with a total volume of 200 micro liter of 30% HP-Î²-CD in PBS. The vehicle-control mice (n = 3) were injected with the same volume of PBS. After 6 h, the inguinal LNs, Liver, and Spleen were removed (within one hour for all samples) and global gene expression was obtained by Affymetrix GeneChip.

2015-02-15 | E-GEOD-63332 | biostudies-arrayexpress

CHEMICAL PROTEOMIC ANALYSIS OF ENDOCANNABINOID HYDROLASE ACTIVITY IN NIEMANN-PICK TYPE C MOUSE BRAIN

Project description:The endocannabinoid system is considered to be an endogenous protective system in various neurodegenerative diseases. Niemann-Pick Type C is a neurodegenerative disease in which the role of the endocannabinoid system has not been studied yet. Here, we report the endocannabinoid hydrolase activity in brain proteomes of a Niemann-Pick type C mouse model as measured by activity-based protein profiling. Diacylglycerol lipase α, α/β-hydrolase domain-containing protein 4 (ABHD4), ABHD6, ABHD12, fatty acid amide hydrolase and monoacylglycerol lipase activities were quantified. Chemical proteomics showed no difference in endocannabinoid hydrolase activity in the brain of wildtype compared to Niemann-Pick C1 protein (NPC1) knockout mice. Three lysosomal serine hydrolases were identified with increased activity in NPC1 knockout mouse brain: retinoid-inducible serine carboxypeptidase, cathepsin A and palmitoyl-protein thioesterase 1, and we conclude that these might be interesting therapeutic targets for future validation studies.

2018-04-19 | PXD008979 | Pride

Global gene expression change in the cerebellum of Niemann-Pick disease type C mice with deletion of Ccl3 or Purkinje neuron-specific NPC1 rescue

Project description:Macrophage inflammatory protein 1alpha/CCL3 protein is a known pro-inflammatory cytokine that can mediate chemotaxis of monocytes and promote cell degranulation. Ccl3 gene expression is elevated in the CNS and visceral tissue of many lysosomal storage disorders. The deletion of Ccl3 in a mouse model of Sandhoff disease was reported to result in reduced monocyte-associated pathology in the brain, delayed neurodegeneration, and prolonged health. However, deletion of Ccl3 in a mouse model of Niemann-Pick C disease was dentrimental or neutral instead of beneficial. Prevention of neuronal loss was instead mediated by providing NPC1 to neurons. We used microarrays to detail the global change in gene expression of the cerebellum in Niemann-Pick C disease animals, Niemann-Pick C disease animals with Ccl3 gene deletion, and Niemann-Pick C disease animals with Purkinje neuron-specific NPC1-YFP rescue.

2012-04-17 | GSE36119 | GEO

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