Inability of α-cyclodextrins to accommodate cholesterol potentially underlies their lack of efficacy and ototoxicity in Niemann-Pick disease type C treatment

Inability of α-cyclodextrins to accommodate cholesterol potentially underlies their lack of efficacy and ototoxicity in Niemann-Pick disease type C treatment

Abstract Niemann-Pick disease type C (NPC) is a life-threatening neurodegenerative disease caused by impaired intracellular cholesterol trafficking. A cyclic heptasaccharide, 2-hydroxypropyl-β-cyclodextrin (HP-β-CD), is currently under clinical investigation for treating NPC, but its ototoxicity rem...

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Main Authors: Yusei Yamada, Mayuko Tanaka, Yusei Ikeda, Yuki Kondo, Toru Takeo, Naomi Nakagata, Toru Miwa, Hiroki Takeda, Yorihisa Orita, Keiichi Motoyama, Taishi Higashi, Hidetoshi Arima, Takahiro Seki, Yuki Kurauchi, Hiroshi Katsuki, Katsumi Higaki, Kotaro Matsusaka, Kentaro Minami, Naoki Yoshikawa, Ryuji Ikeda, Muneaki Matsuo, Tetsumi Irie, Yoichi Ishitsuka
Format: Article
Language:English
Published: Nature Portfolio 2025-08-01
Series:Scientific Reports
Subjects:
Niemann-Pick disease type C
Cholesterol
Cyclodextrin
Molecular docking
Intracerebroventricular administration
Ototoxicity
Online Access:https://doi.org/10.1038/s41598-025-15599-0
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https://doi.org/10.1038/s41598-025-15599-0

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