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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

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Niemann-Pick type C fibroblast analysis

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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.

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

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Niemann-Pick type C fibroblast analysis

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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.

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

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