In vivo Efficacy and Safety Evaluation of Lactosyl-β-cyclodextrin as a Therapeutic Agent for Hepatomegaly in Niemann-Pick Type C Disease

In vivo Efficacy and Safety Evaluation of Lactosyl-β-cyclodextrin as a Therapeutic Agent for Hepatomegaly in Niemann-Pick Type C Disease

Niemann-Pick type C disease (NPC) is a fatal, autosomal recessive disorder, which causes excessive accumulation of free cholesterol in endolysosomes, resulting in progressive hepatomegaly and neurodegeneration. Currently, 2-hydroxypropyl-β-cyclodextrin (HP-β-CyD) is used at a high...

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Bibliographic Details
Main Authors: Yuki Maeda, Keiichi Motoyama, Rena Nishiyama, Taishi Higashi, Risako Onodera, Hideaki Nakamura, Toru Takeo, Naomi Nakagata, Yusei Yamada, Yoichi Ishitsuka, Yuki Kondo, Tetsumi Irie, Takumi Era, Hidetoshi Arima
Format: Article
Language:English
Published: MDPI AG 2019-05-01
Series:Nanomaterials
Subjects:
Cyclodextrins
cholesterol
Niemann-Pick Type C disease
hepatomegaly
liver targeting
Online Access:https://www.mdpi.com/2079-4991/9/5/802

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