Efficacy of 2-Hydroxypropyl-β-cyclodextrin in Niemann–Pick Disease Type C Model Mice and Its Pharmacokinetic Analysis in a Patient with the Disease-Reference-Cited by-同舟云学术

Efficacy of 2-Hydroxypropyl-β-cyclodextrin in Niemann–Pick Disease Type C Model Mice and Its Pharmacokinetic Analysis in a Patient with the Disease

Published:2015 Issue:6 Volume:38 Page:844-851
ISSN:0918-6158
Container-title:Biological & Pharmaceutical Bulletin
language:en
Short-container-title:Biol. Pharm. Bull.

Author:

Tanaka Yuta¹,Yamada Yusei¹,Ishitsuka Yoichi¹,Matsuo Muneaki²,Shiraishi Koki¹,Wada Koki³,Uchio Yushiro¹,Kondo Yuki¹,Takeo Toru⁴,Nakagata Naomi⁴,Higashi Taishi⁵,Motoyama Keiichi⁵,Arima Hidetoshi⁵,Mochinaga Sakiko⁶,Higaki Katsumi⁷,Ohno Kousaku⁸,Irie Tetsumi¹

Affiliation:

1. Department of Clinical Chemistry and Informatics, Graduate School of Pharmaceutical Sciences, Kumamoto University

2. Department of Pediatrics, Faculty of Medicine, Saga University

3. Research Institute, Nihon Shokuhin Kako Co., Ltd.

4. Division of Reproductive Engineering, Center for Animal Resources and Development (CARD), Kumamoto University

5. Department of Physical Pharmaceutics, Graduate School of Pharmaceutical Sciences, Kumamoto University

6. Department of Pharmacy, Faculty of Medicine, Saga University

7. Division of Functional Genomics, Research Center for Bioscience and Technology, Faculty of Medicine, Tottori University

8. Sanin Rosai Hospital

Publisher

Pharmaceutical Society of Japan

Subject

Pharmaceutical Science,Pharmacology,General Medicine

Link

https://www.jstage.jst.go.jp/article/bpb/38/6/38_b14-00726/_pdf

Reference25 articles.

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2. 2) Naureckiene S, Sleat DE, Lackland H, Fensom A, Vanier MT, Wattiaux R, Jadot M, Lobel P. Identification of HE1 as the second gene of Niemann–Pick C disease. Science, 290, 2298–2301 (2000).

3. 3) Vanier MT. Niemann–Pick disease type C. Orphanet J. Rare Dis., 5, 16 (2010).

4. 4) Matsuo M, Togawa M, Hirabaru K, Mochinaga S, Narita A, Adachi M, Egashira M, Irie T, Ohno K. Effects of cyclodextrin in two patients with Niemann–Pick type C disease. Mol. Genet. Metab., 108, 76–81 (2013).

5. 5) Wojtanik KM, Liscum L. The transport of low density lipoprotein-derived cholesterol to the plasma membrane is defective in NPC1 cells. J. Biol. Chem., 278, 14850–14856 (2003).

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