Drug discovery to treat Parkinson's disease using gba1 knock-out medaka

2021 Fiscal Year Final Research Report

• Project/Area Number: 19K17005
• Research Category: Grant-in-Aid for Early-Career Scientists
• Allocation Type: Multi-year Fund
• Review Section: Basic Section 52020:Neurology-related
• Research Institution: Kyoto University
• Principal Investigator: Nakanishi Etsuro 京都大学, 医学研究科, 助教 (40828935)
• Project Period (FY): 2019-04-01 – 2022-03-31
• Keywords: パーキンソン病 / ゴーシェ病 / GBA1 / αシヌクレイン

Outline of Final Research Achievements

This study aims to elucidate the pathogenesis of Parkinson's disease and develop disease-modifying drugs by using GBA1(-/-) medaka, which exhibits endogenous α-synuclein accumulation and neuronal cell loss, as a Parkinoson's disease model animal. To screen the efficacy of the compounds, GBA1(-/-) medaka were treated with the compounds and their effects on survival were evaluated. Six compounds were evaluated during the study period. Biochemical analysis was performed on the compounds that showed an effect on survival rate.

Free Research Field

神経変性疾患

Academic Significance and Societal Importance of the Research Achievements

パーキンソン病および神経型ゴーシェ病、いずれの疾患も現在までに疾患修飾治療薬は実用化されていない。GBA1(-/-)メダカはこれらの疾患の疾患修飾治療薬探索のための有用なモモデル動物であり、本研究で生存期間の延長を見出した化合物は、これらの疾患の疾患修飾治療薬の候補になると考えられる。