PMID-sentid Pub_year Sent_text comp_official_name comp_offsetprotein_name organism prot_offset 34592985-9 2021 Cholesterol storage, mitochondrial dysfunction, and axonal trafficking defects can be ameliorated by treatment with 2-hydroxypropyl-beta-cyclodextrin, a drug that has shown efficacy in NPC1 preclinical models and in a phase 1/2a trial. 2-Hydroxypropyl-beta-cyclodextrin 116-149 NPC intracellular cholesterol transporter 1 Homo sapiens 185-189 32890578-8 2020 These findings have implications for systemic 2HPbetaCD treatment in NPC1 patients in view of the purportedly low levels of SOAT2 activity in human liver. 2-Hydroxypropyl-beta-cyclodextrin 46-55 NPC intracellular cholesterol transporter 1 Homo sapiens 69-73 34296265-4 2021 This study analyzed RNA-Seq data from 42 NPC1 patient-derived, primary fibroblast cell lines to determine transcriptional changes induced by treatment with 2-hydroxypropyl-beta-cyclodextrin (HPbetaCD), a compound currently under investigation in clinical trials. 2-Hydroxypropyl-beta-cyclodextrin 191-199 NPC intracellular cholesterol transporter 1 Homo sapiens 41-45 34296265-8 2021 Overall, this large NPC1 patient-derived dataset provides a comprehensive foundation for understanding the genomic response to HPbetaCD treatment. 2-Hydroxypropyl-beta-cyclodextrin 127-135 NPC intracellular cholesterol transporter 1 Homo sapiens 20-24 28155026-3 2017 The investigational use of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD) showed a promising role in treating NP-C, although efficacy and safety have not been established. 2-Hydroxypropyl-beta-cyclodextrin 27-60 NPC intracellular cholesterol transporter 1 Homo sapiens 110-114 29617956-5 2018 Treatment with 2-hydroxypropyl-beta-cyclodextrin (HPbetaCD), a drug currently being studied in a phase 2b/3 clinical trial, reversed all microglia-associated defects in Npc1-/- animals. 2-Hydroxypropyl-beta-cyclodextrin 15-48 NPC intracellular cholesterol transporter 1 Homo sapiens 169-173 29617956-5 2018 Treatment with 2-hydroxypropyl-beta-cyclodextrin (HPbetaCD), a drug currently being studied in a phase 2b/3 clinical trial, reversed all microglia-associated defects in Npc1-/- animals. 2-Hydroxypropyl-beta-cyclodextrin 50-58 NPC intracellular cholesterol transporter 1 Homo sapiens 169-173 29390035-0 2018 Correction: Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1. 2-Hydroxypropyl-beta-cyclodextrin 32-64 NPC intracellular cholesterol transporter 1 Homo sapiens 90-118 28803710-4 2017 METHODS: In this open-label, dose-escalation phase 1-2a study, we gave monthly intrathecal HPbetaCD to participants with NPC1 with neurological manifestation at the National Institutes of Health (NIH), Bethesda, MD, USA. 2-Hydroxypropyl-beta-cyclodextrin 91-99 NPC intracellular cholesterol transporter 1 Homo sapiens 121-125 28155026-16 2017 HP-beta-CD could help stabilize NP-C with low toxicity potential, although some AEs have been reported. 2-Hydroxypropyl-beta-cyclodextrin 0-10 NPC intracellular cholesterol transporter 1 Homo sapiens 32-36 28155026-17 2017 Moreover, controlled clinical trials would be necessary to evaluate the role of HP-beta-CD in NP-C. 2-Hydroxypropyl-beta-cyclodextrin 80-90 NPC intracellular cholesterol transporter 1 Homo sapiens 94-98 28155026-3 2017 The investigational use of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD) showed a promising role in treating NP-C, although efficacy and safety have not been established. 2-Hydroxypropyl-beta-cyclodextrin 62-72 NPC intracellular cholesterol transporter 1 Homo sapiens 110-114 26664628-2 2015 Currently, 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CyD) has been applied for the treatment of NPC. 2-Hydroxypropyl-beta-cyclodextrin 11-44 NPC intracellular cholesterol transporter 1 Homo sapiens 97-100 28414792-0 2017 Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick Disease type C1. 2-Hydroxypropyl-beta-cyclodextrin 20-52 NPC intracellular cholesterol transporter 1 Homo sapiens 78-106 28414792-1 2017 2-Hydroxypropyl-beta-cyclodextrin (HPbetaCD) has gained recent attention as a potential therapeutic intervention in the treatment of the rare autosomal-recessive, neurodegenerative lysosomal storage disorder Niemann-Pick Disease Type C1 (NPC1). 2-Hydroxypropyl-beta-cyclodextrin 0-33 NPC intracellular cholesterol transporter 1 Homo sapiens 238-242 28414792-1 2017 2-Hydroxypropyl-beta-cyclodextrin (HPbetaCD) has gained recent attention as a potential therapeutic intervention in the treatment of the rare autosomal-recessive, neurodegenerative lysosomal storage disorder Niemann-Pick Disease Type C1 (NPC1). 2-Hydroxypropyl-beta-cyclodextrin 35-43 NPC intracellular cholesterol transporter 1 Homo sapiens 238-242 28179943-2 2017 Currently, treatment of NPC involves the use of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD). 2-Hydroxypropyl-beta-cyclodextrin 48-81 NPC intracellular cholesterol transporter 1 Homo sapiens 24-27 28179943-2 2017 Currently, treatment of NPC involves the use of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD). 2-Hydroxypropyl-beta-cyclodextrin 83-93 NPC intracellular cholesterol transporter 1 Homo sapiens 24-27 28179943-4 2017 One of the methods to reduce the required dose of HP-beta-CD for treatment of NPC is to actively targeting hepatocytes with beta-cyclodextrin (beta-CD). 2-Hydroxypropyl-beta-cyclodextrin 50-60 NPC intracellular cholesterol transporter 1 Homo sapiens 78-81 27572704-1 2016 2-Hydroxy-propyl-beta-cyclodextrin (HPbetaCD), a cholesterol scavenger, is currently undergoing Phase 2b/3 clinical trial for treatment of Niemann Pick Type C-1 (NPC1), a fatal neurodegenerative disorder that stems from abnormal cholesterol accumulation in the endo/lysosomes. 2-Hydroxypropyl-beta-cyclodextrin 36-44 NPC intracellular cholesterol transporter 1 Homo sapiens 139-160 27572704-1 2016 2-Hydroxy-propyl-beta-cyclodextrin (HPbetaCD), a cholesterol scavenger, is currently undergoing Phase 2b/3 clinical trial for treatment of Niemann Pick Type C-1 (NPC1), a fatal neurodegenerative disorder that stems from abnormal cholesterol accumulation in the endo/lysosomes. 2-Hydroxypropyl-beta-cyclodextrin 36-44 NPC intracellular cholesterol transporter 1 Homo sapiens 162-166 26664628-2 2015 Currently, 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CyD) has been applied for the treatment of NPC. 2-Hydroxypropyl-beta-cyclodextrin 46-57 NPC intracellular cholesterol transporter 1 Homo sapiens 97-100 26664628-3 2015 HP-beta-CyD improved hepatosplenomegaly in NPC patients, however, a high dose of HP-beta-CyD was necessary. 2-Hydroxypropyl-beta-cyclodextrin 0-11 NPC intracellular cholesterol transporter 1 Homo sapiens 43-46 24907126-7 2014 In addition, we found that hydroxypropyl-beta-cyclodextrin is much more potent and efficacious in the NPC1 neural stem cells compared to the NPC1 fibroblasts. 2-Hydroxypropyl-beta-cyclodextrin 27-58 NPC intracellular cholesterol transporter 1 Homo sapiens 102-106 26392920-8 2015 However, the unexpected effect of CDX on the GABAergic synaptic transmission is of special interest as a disturbance plays, for example, a crucial role in epilepsy and, moreover, as CDX is currently under investigation as a treatment for NPC1 in humans. 2-Hydroxypropyl-beta-cyclodextrin 182-185 NPC intracellular cholesterol transporter 1 Homo sapiens 238-242 24964810-6 2014 Upon direct CNS delivery of HP-beta-CD, we found increases in plasma 24(S)-HC in two independent NPC1 disease animal models, findings that were confirmed in human NPC1 subjects receiving HP-beta-CD. 2-Hydroxypropyl-beta-cyclodextrin 28-38 NPC intracellular cholesterol transporter 1 Homo sapiens 97-101 24868096-6 2014 The LC-MS/MS methods are ~100-fold more sensitive than the current HPLC assay, and were successfully employed to analyze HP-beta-CD in human plasma and CSF samples to support the phase 1 clinical trial of HP-beta-CD in NPC1 patients. 2-Hydroxypropyl-beta-cyclodextrin 205-215 NPC intracellular cholesterol transporter 1 Homo sapiens 219-223 24283970-2 2014 This paper describes one of the first TRND programs, the development of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD) for the treatment of Niemann-Pick disease type C1 (NPC1). 2-Hydroxypropyl-beta-cyclodextrin 72-105 NPC intracellular cholesterol transporter 1 Homo sapiens 140-168 24283970-2 2014 This paper describes one of the first TRND programs, the development of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD) for the treatment of Niemann-Pick disease type C1 (NPC1). 2-Hydroxypropyl-beta-cyclodextrin 72-105 NPC intracellular cholesterol transporter 1 Homo sapiens 170-174 24283970-2 2014 This paper describes one of the first TRND programs, the development of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD) for the treatment of Niemann-Pick disease type C1 (NPC1). 2-Hydroxypropyl-beta-cyclodextrin 107-117 NPC intracellular cholesterol transporter 1 Homo sapiens 140-168 24283970-2 2014 This paper describes one of the first TRND programs, the development of 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD) for the treatment of Niemann-Pick disease type C1 (NPC1). 2-Hydroxypropyl-beta-cyclodextrin 107-117 NPC intracellular cholesterol transporter 1 Homo sapiens 170-174 23881911-9 2013 Similar alterations were observed in the CSF from the NPC1 feline model following HP-beta-CD treatment. 2-Hydroxypropyl-beta-cyclodextrin 82-92 NPC intracellular cholesterol transporter 1 Homo sapiens 54-58 23881911-5 2013 These lipids were modestly decreased following miglustat treatment, but markedly decreased in response to treatment with 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD), two drugs that have shown efficacy in NPC1 animal models. 2-Hydroxypropyl-beta-cyclodextrin 121-154 NPC intracellular cholesterol transporter 1 Homo sapiens 207-211 23881911-5 2013 These lipids were modestly decreased following miglustat treatment, but markedly decreased in response to treatment with 2-hydroxypropyl-beta-cyclodextrin (HP-beta-CD), two drugs that have shown efficacy in NPC1 animal models. 2-Hydroxypropyl-beta-cyclodextrin 156-166 NPC intracellular cholesterol transporter 1 Homo sapiens 207-211 19884502-6 2009 The NPC1 or NPC2 block in cholesterol delivery to the ER can be overcome by 2-hydroxypropyl-beta-cyclodextrin, which leads to a marked increase in ACAT-mediated cholesterol esterification. 2-Hydroxypropyl-beta-cyclodextrin 76-109 NPC intracellular cholesterol transporter 1 Homo sapiens 4-8