Therapeutic target for enzymatic activity of granular osmiophilic material in CADASIL

2019 Fiscal Year Final Research Report

• Project/Area Number: 17K09760
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Research Field: Neurology
• Research Institution: Kumamoto University
• Principal Investigator: Akihiko Ueda 熊本大学, 病院, 助教 (30613525)
• Project Period (FY): 2017-04-01 – 2020-03-31
• Keywords: CADASIL / 病原性蓄積物 / 酵素阻害薬

Outline of Final Research Achievements

Enzymatic proteins activate chemical reactions of cofactors. The chemical reactions of cofactors are common, while enzymatic proteins are various. Thus, chemical reactions in cofactors are suitable to therapeutic targets. We hypothesized that granular osmiophilic material (GOM) in cerebral autosomal dominant arteriopathy with subcortical infarcts and leukoencephalopathy (CADASIL) have enzymatic activity. To elucidate the hypothesis, we investigate the effect of enzymatic inhibitors on CADASIL model mice. The enzymatic inhibitor was effective to CADASIL model mice. However, it was harmful to inhibit increasing body weight of young mice.

Free Research Field

脳神経内科

Academic Significance and Societal Importance of the Research Achievements

根治的治療法のない遺伝性疾患に対して、治療法開発の糸口をつかむことができた。本研究を基盤として、実際に臨床応用を目指して、治療薬の適切な投与方法や投与量を検討する必要がある。