**Author details**

Juan Eduardo Megías-Vericat\*, María José Company-Albir, Ana Alejandra García-Robles and José Luis Poveda

[11] Yergey AL, Blank PS, Cologna SM, Backlund PS, Porter FD, Darling AJ. Characterization of hydroxypropyl-beta-cyclodextrins used in the treatment of Niemann-Pick disease

Use of 2-Hydroxypropyl-Beta-Cyclodextrin for Niemann-Pick Type C Disease

http://dx.doi.org/10.5772/intechopen.71970

113

type C1. PLoS One. 2017;**12**:e0175478. DOI: doi.org/10.1371/journal.pone.0175478 [12] Kilsdonk EPC, Yancey PG, Stoudt GW, Bangerter FW, Johnson WJ, Phillips MC, et al. Cellular cholesterol efflux mediated by cyclodextrins. The Journal of Biological

[13] Abi-Mosleh L, Infante RE, Radhakrishnan A, Goldstein JL, Brown MS. Cyclodextrin overcomes deficient lysosome-to-endoplasmic reticulum transport of cholesterol in Niemann-Pick type C cells. PNAS. 2009;**106**:19316-19321. DOI: 10.1073/pnas.0910916106

[14] Rosenbaum AI, Zhang G, Warren JD, Maxfield FR. Endocytosis of betacyclodextrins is responsible for cholesterol reduction in Niemann-Pick type C mutant cells. Proceedings of the National Academy of Sciences of the United States of America. 2010;**107**:5477-

[15] Griffin LD, Gong W, Verot L, Mellon SH. Niemann-Pick type C disease involves disrupted neurosteroidogenesis and responds to allopregnanolone. Nature Medicine.

[16] Davidson CD, Ali NF, Micsenyi MC, Stephney G, Renault S, Dobrenis K, et al. Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression. PLoS One. 2009;**4**:e6951.

[17] Liu B, Li H, Repa JJ, Turley SD, Dietschy JM. Genetic variations and treatments that affect the lifespan of the NPC1 mouse. Journal of Lipid Research. 2008;**49**:663-669. DOI:

[18] Camargo F, Erickson RP, Garver WS, Hossain GS, Carbone PN, Heidenreich RA, et al. Cyclodextrins in the treatment of a mouse model of Niemann-Pick C disease. Life

[19] Ward S, O'Donnell P, Fernandez S. Vite CH2-hydroxypropyl-beta-cyclodextrin raises hearing threshold in normal cats and in cats with Niemann-Pick type C disease. Pediatric

[20] Lopez AM, Terpack SJ, Posey KS, Liu B, Ramirez CM, Turley SD. Systemic administration of 2-hydroxypropyl-β-cyclodextrin to symptomatic Npc1-deficient mice slows cholesterol sequestration in the major organs and improves liver function. Clinical and Experimental

[21] Pontikis CC, Davidson CD, Walkley SU, Platt FM, Begley DJ. Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood-brain barrier permeability. Journal of Inherited Metabolic Disease. 2013;**36**:491-

[22] Monnaert V, Tilloy S, Bricout H, Fenart L, Cecchelli R, Monflier E. Behavior of α-, β-, and γ-cyclodextrins and their derivatives on an in vitro model of blood-brain barrier. The Journal of Pharmacology and Experimental Therapeutics. 2004;**310**:745-751. DOI:

Pharmacology & Physiology. 2014;**41**:780-787. DOI: 10.1111/1440-1681.12285

Sciences. 2001;**70**:131-142. DOI: 10.1016/S0024-3205(01)01384-4

Research. 2010;**68**:52-56. DOI: 10.1203/00006450-201011001-00099

Chemistry. 1995;**270**:17250-17256. DOI: 10.1074/jbc.270.29.17250

5482. DOI: 10.1073/pnas.0914309107

2004;**10**:704-711. DOI: 10.1038/nm1073

DOI: 10.1371/journal.pone.0006951

498. DOI: 10.1007/s10545-012-9583-x

10.1124/jpet.104.067512

10.1194/jlr.M700525-JLR200

\*Address all correspondence to: megias\_jua@gva.es

Servicio de Farmacia, Área del Medicamento, Hospital Universitari i Politècnic La Fe, Valencia, Spain
