Therapeutic approach to lysosomal storage disorders by brain-targetable supramolecular prodrugs

2016 Fiscal Year Final Research Report

• Project/Area Number: 26750155
• Research Category: Grant-in-Aid for Young Scientists (B)
• Allocation Type: Multi-year Fund
• Research Field: Biomedical engineering/Biomaterial science and engineering
• Research Institution: Tokyo Medical and Dental University
• Principal Investigator: Tamura Atsushi 東京医科歯科大学, 生体材料工学研究所, 助教 (80631150)
• Project Period (FY): 2014-04-01 – 2017-03-31
• Keywords: ポリロタキサン / シクロデキストリン / コレステロール / ライソゾーム病 / ニーマンピック病C型 / オートファジー

Outline of Final Research Achievements

In this study, stimuli-labile polyrotaxanes containing β-cyclodextrins were developed for the treatment of Niemann-Pick type C (NPC) disease. The administration of the polyrotaxane significantly prolonged the life span of the mouse model of NPC disease at lower dose than clinically tested β-cyclodextrin derivatives. Accordingly, the polyrotaxanes are promising candidate for the treatment of NPC disease.

Free Research Field

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