Devlopment of a novel allosteric chaperone therapy for genetic diseases.

2015 Fiscal Year Final Research Report

• Project/Area Number: 25293230
• Research Category: Grant-in-Aid for Scientific Research (B)
• Allocation Type: Partial Multi-year Fund
• Section: 一般
• Research Field: Pediatrics
• Research Institution: Tottori University
• Principal Investigator: NANBA EIJI 鳥取大学, 生命機能研究支援センター, 教授 (40237631)
• Co-Investigator(Kenkyū-buntansha): KAWATA YASUSHI 国立大学法人鳥取大学, 工学研究科, 教授 (40177697) ; HIGAKI KATSUMI 国立大学法人鳥取大学, 生命機能研究支援センター, 准教授 (90294321)
• Project Period (FY): 2013-04-01 – 2016-03-31
• Keywords: 先天代謝異常症 / ライソゾーム病 / アロステリックシャペロン / 脳疾患 / 治療法開発

Outline of Final Research Achievements

In this study, we have explored to develop non-inhibitory chaperone compounds for human beta-galactosidase deficiency. Screening of the compound library found two candidates, which showed stabilization activity to beta-galactosidase enzyme in vitro without substrate competitive inhibition activity. We also identified a new chaperone candidate, conduramine-F4 based on a lead compound, NOEV. This compound showed a decreased inhibitory activity and increased enzyme enhancing activity against human beta-galactosidase in vitro, indidated as a new chaperone candidate for this disease.

Free Research Field

小児神経学