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Screening for Niemann-Pick type C disease in neurodegenerative diseases.

Boenzi S, Dardis A, Russo P, Bellofatto M, Imbriglio T, Fico T, De Michele G, De Rosa A.

J Clin Neurosci. 2019 Jun 17. pii: S0967-5868(19)30732-5. doi: 10.1016/j.jocn.2019.06.025. [Epub ahead of print]


N-Acyl-O-phosphocholineserines: structures of a novel class of lipids that are biomarkers for Niemann-Pick C1 disease.

Sidhu R, Mondjinou Y, Qian M, Song H, Kumar AB, Hong X, Hsu FF, Dietzen DJ, Yanjanin NM, Porter FD, Berry-Kravis E, Vite CH, Gelb MH, Schaffer JE, Ory DS, Jiang X.

J Lipid Res. 2019 Jun 14. pii: jlr.RA119000157. doi: 10.1194/jlr.RA119000157. [Epub ahead of print]


Laboratory diagnosis of the Niemann-Pick type C disease: an inherited neurodegenerative disorder of cholesterol metabolism.

Sitarska D, Ługowska A.

Metab Brain Dis. 2019 Jun 13. doi: 10.1007/s11011-019-00445-w. [Epub ahead of print] Review.


Long-Term Neuropsychological Outcomes from an Open-Label Phase I/IIa Trial of 2-Hydroxypropyl-β-Cyclodextrins (VTS-270) in Niemann-Pick Disease, Type C1.

Farmer CA, Thurm A, Farhat N, Bianconi S, Keener LA, Porter FD.

CNS Drugs. 2019 Jun 11. doi: 10.1007/s40263-019-00642-2. [Epub ahead of print]


Anomalies in Dopamine Transporter Expression and Primary Cilium Distribution in the Dorsal Striatum of a Mouse Model of Niemann-Pick C1 Disease.

Lucarelli M, Di Pietro C, La Sala G, Fiorenza MT, Marazziti D, Canterini S.

Front Cell Neurosci. 2019 May 24;13:226. doi: 10.3389/fncel.2019.00226. eCollection 2019.


Niemann-Pick Type C Disease Reveals a Link between Lysosomal Cholesterol and PtdIns(4,5)P2 That Regulates Neuronal Excitability.

Vivas O, Tiscione SA, Dixon RE, Ory DS, Dickson EJ.

Cell Rep. 2019 May 28;27(9):2636-2648.e4. doi: 10.1016/j.celrep.2019.04.099.


Characterization of the Subventricular-Thalamo-Cortical Circuit in the NP-C Mouse Brain, and New Insights Regarding Treatment.

Park MH, Choi BJ, Jeong MS, Lee JY, Jung IK, Park KH, Lee HW, Yamaguchi T, Marti HH, Lee BH, Schuchman EH, Jin HK, Bae JS.

Mol Ther. 2019 May 16. pii: S1525-0016(19)30222-9. doi: 10.1016/j.ymthe.2019.05.008. [Epub ahead of print]


In vivo Efficacy and Safety Evaluation of Lactosyl-β-cyclodextrin as a Therapeutic Agent for Hepatomegaly in Niemann-Pick Type C Disease.

Maeda Y, Motoyama K, Nishiyama R, Higashi T, Onodera R, Nakamura H, Takeo T, Nakagata N, Yamada Y, Ishitsuka Y, Kondo Y, Irie T, Era T, Arima H.

Nanomaterials (Basel). 2019 May 25;9(5). pii: E802. doi: 10.3390/nano9050802.


Anti-Niemann Pick C1 Single-Stranded Oligonucleotides with Locked Nucleic Acids Potently Reduce Ebola Virus Infection In Vitro.

Sadewasser A, Dietzel E, Michel S, Klüver M, Helfer M, Thelemann T, Klar R, Eickmann M, Becker S, Jaschinski F.

Mol Ther Nucleic Acids. 2019 Jun 7;16:686-697. doi: 10.1016/j.omtn.2019.04.018. Epub 2019 Apr 25.


Drug-induced increase in lysobisphosphatidic acid reduces the cholesterol overload in Niemann-Pick type C cells and mice.

Moreau D, Vacca F, Vossio S, Scott C, Colaco A, Paz Montoya J, Ferguson C, Damme M, Moniatte M, Parton RG, Platt FM, Gruenberg J.

EMBO Rep. 2019 May 22. pii: e47055. doi: 10.15252/embr.201847055. [Epub ahead of print]


Insights into the Molecular Mechanisms of Cholesterol Binding to the NPC1 and NPC2 Proteins.

Cologna SM, Rosenhouse-Dantsker A.

Adv Exp Med Biol. 2019;1135:139-160. doi: 10.1007/978-3-030-14265-0_8.


[Lipid regulation mechanism and dosage form selection of Xixian Tongshuan Preparation based on molecular simulation methods].

Luo YZ, Zhao BW, Chen X, Gu Y, Zhang YL.

Zhongguo Zhong Yao Za Zhi. 2019 Apr;44(7):1436-1441. doi: 10.19540/j.cnki.cjcmm.20190111.004. Chinese.


Interferon downstream signaling is activated early in pre-symptomatic Niemann-Pick disease type C.

Shin SD, Shin A, Mayagoitia K, Wilson CG, Bellinger DL, Soriano S.

Neurosci Lett. 2019 Jul 27;706:43-50. doi: 10.1016/j.neulet.2019.05.005. Epub 2019 May 5.


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 Apr 30. pii: S1388-1981(19)30064-2. doi: 10.1016/j.bbalip.2019.04.011. [Epub ahead of print]


Macrocyclic Compounds for Drug and Gene Delivery in Immune-Modulating Therapy.

Bai H, Wang J, Li Z, Tang G.

Int J Mol Sci. 2019 Apr 28;20(9). pii: E2097. doi: 10.3390/ijms20092097. Review.


Cholesterol Trafficking: An Emerging Therapeutic Target for Angiogenesis and Cancer.

Lyu J, Yang EJ, Shim JS.

Cells. 2019 Apr 28;8(5). pii: E389. doi: 10.3390/cells8050389. Review.


[Niemann-Pick disease type C caused by NPC1 mutation in a case].

Zhang G, Yu F, Zhang K, Li F, Lyu Y, Gao M, Gai Z, Liu Y.

Zhonghua Yi Xue Yi Chuan Xue Za Zhi. 2019 May 10;36(5):480-483. doi: 10.3760/cma.j.issn.1003-9406.2019.05.016. Chinese.


NPC1-deficient neurons are selectively vulnerable for statin treatment.

Meske V, Albert F, Gerstenberg S, Kallwellis K, Ohm TG.

Neuropharmacology. 2019 Jun;151:159-170. doi: 10.1016/j.neuropharm.2019.04.012. Epub 2019 Apr 17.


Computational Tools Unravel Putative Sterol Binding Sites in the Lysosomal NPC1 Protein.

Elghobashi-Meinhardt N.

J Chem Inf Model. 2019 May 28;59(5):2432-2441. doi: 10.1021/acs.jcim.9b00186. Epub 2019 Apr 15.


Hepatic and neuronal phenotype of NPC1-/- mice.

Santiago-Mujica E, Flunkert S, Rabl R, Neddens J, Loeffler T, Hutter-Paier B.

Heliyon. 2019 Mar 14;5(3):e01293. doi: 10.1016/j.heliyon.2019.e01293. eCollection 2019 Mar.

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