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.

1. 1) Carstea ED, Morris JA, Coleman KG, Loftus SK, Zhang D, Cummings C, Gu J, Rosenfeld MA, Pavan WJ, Krizman DB, Nagle J, Polymeropoulos MH, Sturley SL, Ioannou YA, Higgins ME, Comly M, Cooney A, Brown A, Kaneski CR, Blanchette-Mackie EJ, Dwyer NK, Neufeld EB, Chang TY, Liscum L, Strauss JF 3rd, Ohno K, Zeigler M, Carmi R, Sokol J, Markie D, O'Neill RR, van Diggelen OP, Elleder M, Patterson MC, Brady RO, Vanier MT, Pentchev PG, Tagle DA. Niemann–Pick C1 disease gene: homology to mediators of cholesterol homeostasis. Science, 277, 228–231 (1997).

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).

Cited by 73 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Intracerebroventricular 2-hydroxypropyl-γ-cyclodextrin alleviates hepatic manifestations without distributing to the liver in a murine model of Niemann–Pick disease type C;Life Sciences;2024-08

2. Differently increased volumes of multiple brain areas in Npc1 mutant mice following various drug treatments;Frontiers in Neuroanatomy;2024-07-16

3. The role of transcription factors in the pathogenesis and therapeutic targeting of vascular diseases;Frontiers in Cardiovascular Medicine;2024-04-30

4. Cyclodextrins as therapeutic drugs for treating lipid metabolism disorders;Obesity Reviews;2024-01-10

5. Integrated multi-omics profiling highlights the benefits of resveratrol hydroxypropyl-β-cyclodextrin inclusion complex for A53T transgenic mice through the microbiota–gut–brain axis;Food & Function;2024