Influence of Npc1 genotype on the toxicity of hydroxypropyl-β-cyclodextrin, a potentially therapeutic agent, in Niemann–Pick Type C disease models-Reference-Cited by-同舟云学术

Influence of Npc1 genotype on the toxicity of hydroxypropyl-β-cyclodextrin, a potentially therapeutic agent, in Niemann–Pick Type C disease models

Published:2014 Issue: Volume:1 Page:19-30
ISSN:2214-4269
Container-title:Molecular Genetics and Metabolism Reports
language:en
Short-container-title:Molecular Genetics and Metabolism Reports

Author:

Tanaka Yuta,Ishitsuka Yoichi,Yamada Yusei,Kondo Yuki,Takeo Toru,Nakagata Naomi,Higashi Taishi,Motoyama Keiichi,Arima Hidetoshi,Matsuo Muneaki,Higaki Katsumi,Ohno Kousaku,Irie Tetsumi

Publisher

Elsevier BV

Subject

Endocrinology,Genetics,Molecular Biology

Reference21 articles.

1. Niemann-Pick disease type C;Vanier;Orphanet J. Rare Dis.,2010

2. Reversal of defective lysosomal transport in NPC disease ameliorates liver dysfunction and neurodegeneration in the npc1−/− mouse;Liu;Proc. Natl. Acad. Sci. U. S. A.,2009

3. Chronic cyclodextrin treatment of murine Niemann-Pick C disease ameliorates neuronal cholesterol and glycosphingolipid storage and disease progression;Davidson;PLoS One,2009

4. Weekly cyclodextrin administration normalizes cholesterol metabolism in nearly every organ of the Niemann-Pick type C1 mouse and markedly prolongs life;Ramirez;Pediatr. Res.,2010

5. Effects of cyclodextrin in two patients with Niemann-Pick Type C disease;Matsuo;Mol. Genet. Metab.,2013

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1. Different solubilizing ability of cyclodextrin derivatives for cholesterol in Niemann–Pick disease type C treatment;Clinical and Translational Medicine;2023-08

2. Cyclodextrins applied to the treatment of lysosomal storage disorders;Advanced Drug Delivery Reviews;2022-12

3. Fine-tuned cholesterol solubilizer, mono-6-O-α-D-maltosyl-γ-cyclodextrin, ameliorates experimental Niemann–Pick disease type C without hearing loss;Biomedicine & Pharmacotherapy;2022-11

4. Inclusion Solves Insolubility —Translational Research Cycle from Bedside to Bench and Bench to Bedside for Drug Development Targeting Niemann-Pick Disease Type C—;YAKUGAKU ZASSHI;2022-04-01

5. Differential mode of cholesterol inclusion with 2‐hydroxypropyl‐cyclodextrins increases safety margin in treatment of Niemann‐Pick disease type C;British Journal of Pharmacology;2021-05-12