Development of the deaminoneuraminic acid therapy for treatment of congenital disorders of glycosylation Ib

2016 Fiscal Year Final Research Report

• Project/Area Number: 15K14918
• Research Category: Grant-in-Aid for Challenging Exploratory Research
• Allocation Type: Multi-year Fund
• Research Field: Applied molecular and cellular biology
• Research Institution: Nagoya University
• Principal Investigator: Kitajima Ken 名古屋大学, 生物機能開発利用研究センター, 教授 (80192558)
• Project Period (FY): 2015-04-01 – 2017-03-31
• Keywords: デアミノノイラミン酸 / 先天性 / マンノースセラピー / 代謝 / 糖質 / 治療 / シアル酸 / 代謝酵素

Outline of Final Research Achievements

The objective of this study is to development of the KDN therapy as a new drug for CDG1b patients. In this disorder, glycan amount is reduced due to a deficiency of Man. The reduced glycans are rescued by the mannose therapy; however, the development of new method would be desired, if possible. In this study, we thus try to demonstrate the effectiveness of the new method. As a result, we attained the establishment of analytical methods for metabolic products and the amount of cell surface glycan chains. On the other hand, CDG1b model cell lines could not be established within the research period, even after many efforts. Notably, we succeeded in demonstrating that KDN is incorporated into cells through ponocytosis as well as through one of monocarboxylate transporters unlike a major sialic acid species Neu5Ac.

Free Research Field

糖鎖生物学