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Lactose-Appended Hydroxypropyl-β-Cyclodextrin Lowers Cholesterol Accumulation and Alleviates Motor Dysfunction in Niemann-Pick Type C Disease Model Mice
Takumi Nishida 1, Ryoma Yokoyama 1, Yuto Kubohira 1, Yuki Maeda 1 2, Toru Takeo 3, Naomi Nakagata 3, Hiroki Takagi 4, Kandai Ishikura 4, Kazunori Yanagihara 4, Shogo Misumi 1, Naoki Kishimoto 1, Yoichi Ishitsuka 1, Yuki Kondo 1, Tetsumi Irie 1 2, Minami Soga 5, Takumi Era 5, Risako Onodera 1, Taishi Higashi 1 6, Keiichi Motoyama 1
...Herein, we have fabricated lactose-appended hydroxypropyl-β-cyclodextrin (Lac-HPβCD) and evaluated its lowering effects on cholesterol accumulation in NPC model mice. We reveal that Lac-HPβCD lowers cholesterol accumulation in the liver and spleen by reducing the amount of free cholesterol. Moreover, Lac-HPβCD reduces the amount of free cholesterol in the cerebrum and slightly alleviates motor dysfunction. These results suggest that Lac-HPβCD has potential for the treatment of NPC.