321 related articles for article (PubMed ID: 24964810)
1. Cholesterol homeostatic responses provide biomarkers for monitoring treatment for the neurodegenerative disease Niemann-Pick C1 (NPC1).
Tortelli B; Fujiwara H; Bagel JH; Zhang J; Sidhu R; Jiang X; Yanjanin NM; Shankar RK; Carillo-Carasco N; Heiss J; Ottinger E; Porter FD; Schaffer JE; Vite CH; Ory DS
Hum Mol Genet; 2014 Nov; 23(22):6022-33. PubMed ID: 24964810
[TBL] [Abstract][Full Text] [Related]
2. Identification of Niemann-Pick C1 disease biomarkers through sphingolipid profiling.
Fan M; Sidhu R; Fujiwara H; Tortelli B; Zhang J; Davidson C; Walkley SU; Bagel JH; Vite C; Yanjanin NM; Porter FD; Schaffer JE; Ory DS
J Lipid Res; 2013 Oct; 54(10):2800-14. PubMed ID: 23881911
[TBL] [Abstract][Full Text] [Related]
3. Differential Effects of 2-Hydroxypropyl-Cyclodextrins on Lipid Accumulation in
Hoque S; Kondo Y; Sakata N; Yamada Y; Fukaura M; Higashi T; Motoyama K; Arima H; Higaki K; Hayashi A; Komiya T; Ishitsuka Y; Irie T
Int J Mol Sci; 2020 Jan; 21(3):. PubMed ID: 32019132
[TBL] [Abstract][Full Text] [Related]
4. Intravenous 2-hydroxypropyl-β-cyclodextrin (Trappsol® Cyclo™) demonstrates biological activity and impacts cholesterol metabolism in the central nervous system and peripheral tissues in adult subjects with Niemann-Pick Disease Type C1: Results of a phase 1 trial.
Hastings C; Liu B; Hurst B; Cox GF; Hrynkow S
Mol Genet Metab; 2022 Dec; 137(4):309-319. PubMed ID: 36279795
[TBL] [Abstract][Full Text] [Related]
5. Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1-2 trial.
Ory DS; Ottinger EA; Farhat NY; King KA; Jiang X; Weissfeld L; Berry-Kravis E; Davidson CD; Bianconi S; Keener LA; Rao R; Soldatos A; Sidhu R; Walters KA; Xu X; Thurm A; Solomon B; Pavan WJ; Machielse BN; Kao M; Silber SA; McKew JC; Brewer CC; Vite CH; Walkley SU; Austin CP; Porter FD
Lancet; 2017 Oct; 390(10104):1758-1768. PubMed ID: 28803710
[TBL] [Abstract][Full Text] [Related]
6. Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression.
Davidson CD; Ali NF; Micsenyi MC; Stephney G; Renault S; Dobrenis K; Ory DS; Vanier MT; Walkley SU
PLoS One; 2009 Sep; 4(9):e6951. PubMed ID: 19750228
[TBL] [Abstract][Full Text] [Related]
7. Intrathecal 2-hydroxypropyl-beta-cyclodextrin in a single patient with Niemann-Pick C1.
Maarup TJ; Chen AH; Porter FD; Farhat NY; Ory DS; Sidhu R; Jiang X; Dickson PI
Mol Genet Metab; 2015; 116(1-2):75-9. PubMed ID: 26189084
[TBL] [Abstract][Full Text] [Related]
8. In Vitro and In Vivo Evaluation of 6-O-α-Maltosyl-β-Cyclodextrin as a Potential Therapeutic Agent Against Niemann-Pick Disease Type C.
Yasmin N; Ishitsuka Y; Fukaura M; Yamada Y; Nakahara S; Ishii A; Kondo Y; Takeo T; Nakagata N; Motoyama K; Higashi T; Okada Y; Nishikawa J; Ichikawa A; Iohara D; Hirayama F; Higaki K; Ohno K; Matsuo M; Irie T
Int J Mol Sci; 2019 Mar; 20(5):. PubMed ID: 30845767
[TBL] [Abstract][Full Text] [Related]
9. Cyclodextrin mediates rapid changes in lipid balance in Npc1-/- mice without carrying cholesterol through the bloodstream.
Taylor AM; Liu B; Mari Y; Liu B; Repa JJ
J Lipid Res; 2012 Nov; 53(11):2331-42. PubMed ID: 22892156
[TBL] [Abstract][Full Text] [Related]
10. Long-term administration of intravenous Trappsol® Cyclo™ (HP-β-CD) results in clinical benefits and stabilization or slowing of disease progression in patients with Niemann-Pick disease type C1: Results of an international 48-week Phase I/II trial.
Sharma R; Hastings C; Staretz-Chacham O; Raiman J; Paucar M; Spiegel R; Murray B; Hurst B; Liu B; Kjems L; Hrynkow S
Mol Genet Metab Rep; 2023 Sep; 36():100988. PubMed ID: 37670901
[TBL] [Abstract][Full Text] [Related]
11. Microarray expression analysis and identification of serum biomarkers for Niemann-Pick disease, type C1.
Cluzeau CV; Watkins-Chow DE; Fu R; Borate B; Yanjanin N; Dail MK; Davidson CD; Walkley SU; Ory DS; Wassif CA; Pavan WJ; Porter FD
Hum Mol Genet; 2012 Aug; 21(16):3632-46. PubMed ID: 22619379
[TBL] [Abstract][Full Text] [Related]
12. Normalization of cholesterol homeostasis by 2-hydroxypropyl-β-cyclodextrin in neurons and glia from Niemann-Pick C1 (NPC1)-deficient mice.
Peake KB; Vance JE
J Biol Chem; 2012 Mar; 287(12):9290-8. PubMed ID: 22277650
[TBL] [Abstract][Full Text] [Related]
13. Collaborative development of 2-hydroxypropyl-β-cyclodextrin for the treatment of Niemann-Pick type C1 disease.
Ottinger EA; Kao ML; Carrillo-Carrasco N; Yanjanin N; Shankar RK; Janssen M; Brewster M; Scott I; Xu X; Cradock J; Terse P; Dehdashti SJ; Marugan J; Zheng W; Portilla L; Hubbs A; Pavan WJ; Heiss J; Vite CH; Walkley SU; Ory DS; Silber SA; Porter FD; Austin CP; McKew JC
Curr Top Med Chem; 2014; 14(3):330-9. PubMed ID: 24283970
[TBL] [Abstract][Full Text] [Related]
14. Investigation of 2-Hydroxypropyl-β-Cyclodextrin Treatment in a Neuronal-Like Cell Model of Niemann-Pick Type C Using Quantitative Proteomics.
Cougnoux A; Pergande MR; Serna-Perez F; Cologna SM
J Am Soc Mass Spectrom; 2023 Apr; 34(4):668-675. PubMed ID: 36920149
[TBL] [Abstract][Full Text] [Related]
15. Differential mode of cholesterol inclusion with 2-hydroxypropyl-cyclodextrins increases safety margin in treatment of Niemann-Pick disease type C.
Yamada Y; Ishitsuka Y; Kondo Y; Nakahara S; Nishiyama A; Takeo T; Nakagata N; Motoyama K; Higashi T; Arima H; Kamei S; Shuto T; Kai H; Hayashino Y; Sugita M; Kikuchi T; Hirata F; Miwa T; Takeda H; Orita Y; Seki T; Ohta T; Kurauchi Y; Katsuki H; Matsuo M; Higaki K; Ohno K; Matsumoto S; Era T; Irie T
Br J Pharmacol; 2021 Jul; 178(13):2727-2746. PubMed ID: 33782944
[TBL] [Abstract][Full Text] [Related]
16. Weekly cyclodextrin administration normalizes cholesterol metabolism in nearly every organ of the Niemann-Pick type C1 mouse and markedly prolongs life.
Ramirez CM; Liu B; Taylor AM; Repa JJ; Burns DK; Weinberg AG; Turley SD; Dietschy JM
Pediatr Res; 2010 Oct; 68(4):309-15. PubMed ID: 20581737
[TBL] [Abstract][Full Text] [Related]
17. Intracerebroventricular Treatment with 2-Hydroxypropyl-β-Cyclodextrin Decreased Cerebellar and Hepatic Glycoprotein Nonmetastatic Melanoma Protein B (GPNMB) Expression in Niemann-Pick Disease Type C Model Mice.
Fukaura M; Ishitsuka Y; Shirakawa S; Ushihama N; Yamada Y; Kondo Y; Takeo T; Nakagata N; Motoyama K; Higashi T; Arima H; Kurauchi Y; Seki T; Katsuki H; Higaki K; Matsuo M; Irie T
Int J Mol Sci; 2021 Jan; 22(1):. PubMed ID: 33466390
[TBL] [Abstract][Full Text] [Related]
18. Synthesis of the Anionic Hydroxypropyl-β-cyclodextrin:Poly(decamethylenephosphate) Polyrotaxane and Evaluation of its Cholesterol Efflux Potential in Niemann-Pick C1 Cells.
Egele K; Samaddar S; Schneider N; Thompson D; Wenz G
J Mater Chem B; 2019 Jan; 7(4):528-537. PubMed ID: 31372225
[TBL] [Abstract][Full Text] [Related]
19. Polyrotaxane-based systemic delivery of β-cyclodextrins for potentiating therapeutic efficacy in a mouse model of Niemann-Pick type C disease.
Tamura A; Yui N
J Control Release; 2018 Jan; 269():148-158. PubMed ID: 29138063
[TBL] [Abstract][Full Text] [Related]
20. Systemic administration of 2-hydroxypropyl-β-cyclodextrin to symptomatic Npc1-deficient mice slows cholesterol sequestration in the major organs and improves liver function.
Lopez AM; Terpack SJ; Posey KS; Liu B; Ramirez CM; Turley SD
Clin Exp Pharmacol Physiol; 2014 Oct; 41(10):780-7. PubMed ID: 25115571
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]
