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Items: 1 to 20 of 103

1.

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. doi: 10.1016/j.bbalip.2019.04.011. Epub 2019 Apr 30.

PMID:
31051283
2.

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. doi: 10.1016/j.ymgme.2018.10.009. Epub 2018 Oct 30.

PMID:
30392741
3.

Chronic administration of an HDAC inhibitor treats both neurological and systemic Niemann-Pick type C disease in a mouse model.

Alam MS, Getz M, Haldar K.

Sci Transl Med. 2016 Feb 17;8(326):326ra23. doi: 10.1126/scitranslmed.aad9407.

PMID:
26888431
4.

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.

5.

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 24;6(1):98. doi: 10.1186/s40478-018-0599-9.

6.

Tolerance of chronic HDACi treatment for neurological, visceral and lung Niemann-Pick Type C disease in mice.

Alam MS, Cooper B, Farris JD, Haldar K.

Sci Rep. 2018 Mar 1;8(1):3875. doi: 10.1038/s41598-018-22162-7.

7.

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 15;643:117-123. doi: 10.1016/j.gene.2017.12.006. Epub 2017 Dec 6.

PMID:
29223359
8.

Normalization of Hepatic Homeostasis in the Npc1nmf164 Mouse Model of Niemann-Pick Type C Disease Treated with the Histone Deacetylase Inhibitor Vorinostat.

Munkacsi AB, Hammond N, Schneider RT, Senanayake DS, Higaki K, Lagutin K, Bloor SJ, Ory DS, Maue RA, Chen FW, Hernandez-Ono A, Dahlson N, Repa JJ, Ginsberg HN, Ioannou YA, Sturley SL.

J Biol Chem. 2017 Mar 17;292(11):4395-4410. doi: 10.1074/jbc.M116.770578. Epub 2016 Dec 28.

9.

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 30;6:31750. doi: 10.1038/srep31750.

10.

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. doi: 10.2174/1381612823666171019164220. Review.

11.

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.

12.

Quantitative Analysis of the Proteome Response to the Histone Deacetylase Inhibitor (HDACi) Vorinostat in Niemann-Pick Type C1 disease.

Subramanian K, Rauniyar N, Lavalleé-Adam M, Yates JR 3rd, Balch WE.

Mol Cell Proteomics. 2017 Nov;16(11):1938-1957. doi: 10.1074/mcp.M116.064949. Epub 2017 Aug 31.

13.

Main Olfactory and Vomeronasal Epithelium Are Differently Affected in Niemann-Pick Disease Type C1.

Witt M, Thiemer R, Meyer A, Schmitt O, Wree A.

Int J Mol Sci. 2018 Nov 12;19(11). pii: E3563. doi: 10.3390/ijms19113563.

14.

Histone deacetylase inhibitors correct the cholesterol storage defect in most Niemann-Pick C1 mutant cells.

Pipalia NH, Subramanian K, Mao S, Ralph H, Hutt DM, Scott SM, Balch WE, Maxfield FR.

J Lipid Res. 2017 Apr;58(4):695-708. doi: 10.1194/jlr.M072140. Epub 2017 Feb 13. Erratum in: J Lipid Res. 2017 Sep;58(9):1932.

15.

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.

16.

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 24;19(4). pii: E972. doi: 10.3390/ijms19040972.

17.

Modeling Niemann-Pick disease type C1 in zebrafish: a robust platform for in vivo screening of candidate therapeutic compounds.

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 15;11(9). pii: dmm034165. doi: 10.1242/dmm.034165.

18.

Methyl-β-cyclodextrin restores impaired autophagy flux in Niemann-Pick C1-deficient cells through activation of AMPK.

Dai S, Dulcey AE, Hu X, Wassif CA, Porter FD, Austin CP, Ory DS, Marugan J, Zheng W.

Autophagy. 2017 Aug 3;13(8):1435-1451. doi: 10.1080/15548627.2017.1329081. Epub 2017 Jun 14.

19.

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 6;20(5). pii: E1152. doi: 10.3390/ijms20051152.

20.

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 1;77(3):229-245. doi: 10.1093/jnen/nlx119.

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