263 related articles for article (PubMed ID: 34296265)
1. Transcriptome of HPβCD-treated Niemann-Pick disease type C1 cells highlights GPNMB as a biomarker for therapeutics.
Rodriguez-Gil JL; Baxter LL; Watkins-Chow DE; Johnson NL; Davidson CD; Carlson SR; Incao AA; ; Wallom KL; Farhat NY; Platt FM; Dale RK; Porter FD; Pavan WJ
Hum Mol Genet; 2021 Nov; 30(24):2456-2468. PubMed ID: 34296265
[TBL] [Abstract][Full Text] [Related]
2. 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]
3. 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]
4. The susceptibility of cochlear outer hair cells to cyclodextrin is not related to their electromotile activity.
Zhou Y; Takahashi S; Homma K; Duan C; Zheng J; Cheatham MA; Zheng J
Acta Neuropathol Commun; 2018 Sep; 6(1):98. PubMed ID: 30249300
[TBL] [Abstract][Full Text] [Related]
5. Necroptosis inhibition as a therapy for Niemann-Pick disease, type C1: Inhibition of RIP kinases and combination therapy with 2-hydroxypropyl-β-cyclodextrin.
Cougnoux A; Clifford S; Salman A; Ng SL; Bertin J; Porter FD
Mol Genet Metab; 2018 Dec; 125(4):345-350. PubMed ID: 30392741
[TBL] [Abstract][Full Text] [Related]
6. 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]
7. LC3 Immunostaining in the Inferior Olivary Nuclei of Cats With Niemann-Pick Disease Type C1 Is Associated With Patterned Purkinje Cell Loss.
Gurda BL; Bagel JH; Fisher SJ; Schultz ML; Lieberman AP; Hand P; Vite CH; Swain GP
J Neuropathol Exp Neurol; 2018 Mar; 77(3):229-245. PubMed ID: 29346563
[TBL] [Abstract][Full Text] [Related]
8. Application of a glycinated bile acid biomarker for diagnosis and assessment of response to treatment in Niemann-pick disease type C1.
Sidhu R; Kell P; Dietzen DJ; Farhat NY; Do AND; Porter FD; Berry-Kravis E; Reunert J; Marquardt T; Giugliani R; Lourenço CM; Wang RY; Movsesyan N; Plummer E; Schaffer JE; Ory DS; Jiang X
Mol Genet Metab; 2020 Dec; 131(4):405-417. PubMed ID: 33257258
[TBL] [Abstract][Full Text] [Related]
9. Genetic background modifies phenotypic severity and longevity in a mouse model of Niemann-Pick disease type C1.
Rodriguez-Gil JL; Watkins-Chow DE; Baxter LL; Elliot G; Harper UL; Wincovitch SM; Wedel JC; Incao AA; Huebecker M; Boehm FJ; Garver WS; Porter FD; Broman KW; Platt FM; Pavan WJ
Dis Model Mech; 2020 Mar; 13(3):. PubMed ID: 31996359
[TBL] [Abstract][Full Text] [Related]
10. Hydroxypropyl-beta and -gamma cyclodextrins rescue cholesterol accumulation in Niemann-Pick C1 mutant cell via lysosome-associated membrane protein 1.
Singhal A; Szente L; Hildreth JEK; Song B
Cell Death Dis; 2018 Oct; 9(10):1019. PubMed ID: 30282967
[TBL] [Abstract][Full Text] [Related]
11. Microglia activation in Niemann-Pick disease, type C1 is amendable to therapeutic intervention.
Cougnoux A; Drummond RA; Collar AL; Iben JR; Salman A; Westgarth H; Wassif CA; Cawley NX; Farhat NY; Ozato K; Lionakis MS; Porter FD
Hum Mol Genet; 2018 Jun; 27(12):2076-2089. PubMed ID: 29617956
[TBL] [Abstract][Full Text] [Related]
12. 2-Hydroxypropyl-β-cyclodextrins and the Blood-Brain Barrier: Considerations for Niemann-Pick Disease Type C1.
Calias P
Curr Pharm Des; 2017; 23(40):6231-6238. PubMed ID: 29065825
[TBL] [Abstract][Full Text] [Related]
13. PEG-lipid micelles enable cholesterol efflux in Niemann-Pick Type C1 disease-based lysosomal storage disorder.
Brown A; Patel S; Ward C; Lorenz A; Ortiz M; DuRoss A; Wieghardt F; Esch A; Otten EG; Heiser LM; Korolchuk VI; Sun C; Sarkar S; Sahay G
Sci Rep; 2016 Aug; 6():31750. PubMed ID: 27572704
[TBL] [Abstract][Full Text] [Related]
14. 2-Hydroxypropyl-β-cyclodextrin is the active component in a triple combination formulation for treatment of Niemann-Pick C1 disease.
Davidson J; Molitor E; Moores S; Gale SE; Subramanian K; Jiang X; Sidhu R; Kell P; Zhang J; Fujiwara H; Davidson C; Helquist P; Melancon BJ; Grigalunas M; Liu G; Salahi F; Wiest O; Xu X; Porter FD; Pipalia NH; Cruz DL; Holson EB; Schaffer JE; Walkley SU; Maxfield FR; Ory DS
Biochim Biophys Acta Mol Cell Biol Lipids; 2019 Oct; 1864(10):1545-1561. PubMed ID: 31051283
[TBL] [Abstract][Full Text] [Related]
15. Evaluation of Two Liver Treatment Strategies in a Mouse Model of Niemann-Pick-Disease Type C1.
Ebner L; Gläser A; Bräuer A; Witt M; Wree A; Rolfs A; Frank M; Vollmar B; Kuhla A
Int J Mol Sci; 2018 Mar; 19(4):. PubMed ID: 29587349
[TBL] [Abstract][Full Text] [Related]
16. 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]
17. Modeling Niemann-Pick disease type C1 in zebrafish: a robust platform for
Tseng WC; Loeb HE; Pei W; Tsai-Morris CH; Xu L; Cluzeau CV; Wassif CA; Feldman B; Burgess SM; Pavan WJ; Porter FD
Dis Model Mech; 2018 Aug; 11(9):. PubMed ID: 30135069
[TBL] [Abstract][Full Text] [Related]
18. In Niemann-Pick C1 mouse models, glial-only expression of the normal gene extends survival much further than do changes in genetic background or treatment with hydroxypropyl-beta-cyclodextrin.
Marshall CA; Watkins-Chow DE; Palladino G; Deutsch G; Chandran K; Pavan WJ; Erickson RP
Gene; 2018 Feb; 643():117-123. PubMed ID: 29223359
[TBL] [Abstract][Full Text] [Related]
19. Susceptibility of outer hair cells to cholesterol chelator 2-hydroxypropyl-β-cyclodextrine is prestin-dependent.
Takahashi S; Homma K; Zhou Y; Nishimura S; Duan C; Chen J; Ahmad A; Cheatham MA; Zheng J
Sci Rep; 2016 Feb; 6():21973. PubMed ID: 26903308
[TBL] [Abstract][Full Text] [Related]
20. Identification of Brain-Specific Treatment Effects in NPC1 Disease by Focusing on Cellular and Molecular Changes of Sphingosine-1-Phosphate Metabolism.
Gläser A; Hammerl F; Gräler MH; Coldewey SM; Völkner C; Frech MJ; Yang F; Luo J; Tönnies E; von Bohlen Und Halbach O; Brandt N; Heimes D; Neßlauer AM; Korenke GC; Owczarek-Lipska M; Neidhardt J; Rolfs A; Wree A; Witt M; Bräuer AU
Int J Mol Sci; 2020 Jun; 21(12):. PubMed ID: 32599915
[TBL] [Abstract][Full Text] [Related]
[Next] [New Search]