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Items: 1 to 20 of 246

1.

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 15;21(16):3632-46. doi: 10.1093/hmg/dds193. Epub 2012 May 22.

2.

Defective Cytochrome P450-Catalysed Drug Metabolism in Niemann-Pick Type C Disease.

Nicoli ER, Al Eisa N, Cluzeau CV, Wassif CA, Gray J, Burkert KR, Smith DA, Morris L, Cologna SM, Peer CJ, Sissung TM, Uscatu CD, Figg WD, Pavan WJ, Vite CH, Porter FD, Platt FM.

PLoS One. 2016 Mar 28;11(3):e0152007. doi: 10.1371/journal.pone.0152007. eCollection 2016.

3.

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 15;23(22):6022-33. doi: 10.1093/hmg/ddu331. Epub 2014 Jun 25.

4.

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. doi: 10.1194/jlr.M040618. Epub 2013 Jul 23.

5.

Evaluation of an anti-tumor necrosis factor therapeutic in a mouse model of Niemann-Pick C liver disease.

Vincent M, Sayre NL, Graham MJ, Crooke RM, Shealy DJ, Liscum L.

PLoS One. 2010 Sep 23;5(9):e12941. doi: 10.1371/journal.pone.0012941.

6.

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 16;287(12):9290-8. doi: 10.1074/jbc.M111.326405. Epub 2012 Jan 25.

7.

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 14;390(10104):1758-1768. doi: 10.1016/S0140-6736(17)31465-4. Epub 2017 Aug 10.

8.

Mutant human APP exacerbates pathology in a mouse model of NPC and its reversal by a β-cyclodextrin.

Maulik M, Ghoshal B, Kim J, Wang Y, Yang J, Westaway D, Kar S.

Hum Mol Genet. 2012 Nov 15;21(22):4857-75. doi: 10.1093/hmg/dds322. Epub 2012 Aug 6.

PMID:
22869680
9.

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. doi: 10.1111/1440-1681.12285.

10.

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. doi: 10.1203/00006450-201011001-00604.

11.

Increased Regenerative Capacity of the Olfactory Epithelium in Niemann-Pick Disease Type C1.

Meyer A, Wree A, Günther R, Holzmann C, Schmitt O, Rolfs A, Witt M.

Int J Mol Sci. 2017 Apr 6;18(4). pii: E777. doi: 10.3390/ijms18040777.

12.

Pulmonary abnormalities in animal models due to Niemann-Pick type C1 (NPC1) or C2 (NPC2) disease.

Roszell BR, Tao JQ, Yu KJ, Gao L, Huang S, Ning Y, Feinstein SI, Vite CH, Bates SR.

PLoS One. 2013 Jul 2;8(7):e67084. doi: 10.1371/journal.pone.0067084. Print 2013.

13.

Genomic expression analyses reveal lysosomal, innate immunity proteins, as disease correlates in murine models of a lysosomal storage disorder.

Alam MS, Getz M, Safeukui I, Yi S, Tamez P, Shin J, Velázquez P, Haldar K.

PLoS One. 2012;7(10):e48273. doi: 10.1371/journal.pone.0048273. Epub 2012 Oct 19.

14.

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 11;4(9):e6951. doi: 10.1371/journal.pone.0006951.

15.

Efficacy of N-acetylcysteine in phenotypic suppression of mouse models of Niemann-Pick disease, type C1.

Fu R, Wassif CA, Yanjanin NM, Watkins-Chow DE, Baxter LL, Incao A, Liscum L, Sidhu R, Firnkes S, Graham M, Ory DS, Porter FD, Pavan WJ.

Hum Mol Genet. 2013 Sep 1;22(17):3508-23. doi: 10.1093/hmg/ddt206. Epub 2013 May 10.

16.

Alteration of gene expression profile in Niemann-Pick type C mice correlates with tissue damage and oxidative stress.

Vázquez MC, del Pozo T, Robledo FA, Carrasco G, Pavez L, Olivares F, González M, Zanlungo S.

PLoS One. 2011;6(12):e28777. doi: 10.1371/journal.pone.0028777. Epub 2011 Dec 22.

17.

Ontogenic changes in lung cholesterol metabolism, lipid content, and histology in mice with Niemann-Pick type C disease.

Ramirez CM, Lopez AM, Le LQ, Posey KS, Weinberg AG, Turley SD.

Biochim Biophys Acta. 2014 Jan;1841(1):54-61. doi: 10.1016/j.bbalip.2013.09.010. Epub 2013 Sep 26.

18.

Olfactory deficits in Niemann-Pick type C1 (NPC1) disease.

Hovakimyan M, Meyer A, Lukas J, Luo J, Gudziol V, Hummel T, Rolfs A, Wree A, Witt M.

PLoS One. 2013 Dec 31;8(12):e82216. doi: 10.1371/journal.pone.0082216. eCollection 2013.

19.

FTY720/fingolimod increases NPC1 and NPC2 expression and reduces cholesterol and sphingolipid accumulation in Niemann-Pick type C mutant fibroblasts.

Newton J, Hait NC, Maceyka M, Colaco A, Maczis M, Wassif CA, Cougnoux A, Porter FD, Milstien S, Platt N, Platt FM, Spiegel S.

FASEB J. 2017 Apr;31(4):1719-1730. doi: 10.1096/fj.201601041R. Epub 2017 Jan 12.

20.

Endocytosis of beta-cyclodextrins is responsible for cholesterol reduction in Niemann-Pick type C mutant cells.

Rosenbaum AI, Zhang G, Warren JD, Maxfield FR.

Proc Natl Acad Sci U S A. 2010 Mar 23;107(12):5477-82. doi: 10.1073/pnas.0914309107. Epub 2010 Mar 8.

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