Disease Modeling and Drug Development for Parkinson’s Disease Using iPS Cells

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  • Akamatsu Wado
    Center for Genomic and Regenerative Medicine, Juntendo University

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  • iPS細胞を用いたパーキンソン病の病態解析と創薬

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Abstract

<p>Parkinson’s disease is a neurodegenerative disorder affecting approximately 1 in 100 people over the age of 60. However, the sporadic cases, which account for 90% of the total cases, are expected to be diverse based on the clinical symptoms. Therefore, stratification of each case and clarification regarding the pathophysiology are essential for developing effective treatments. We have established and analyzed various types of Parkinson’s disease iPS cells. Further, we have recently screened 320 compounds using midbrain-specific neurons differentiated from iPS cells obtained from patients with familial Parkinson’s disease showing abnormal mitochondrial clearance. We successfully identified four compounds that could restore the disease phenotypes and cellular vulnerability. The candidate compounds also showed phenotypic restoring effects in Drosophila models and sporadic iPS cells. Thus, in Parkinson’s disease, drug discovery using iPS cells derived from rare familial cases may lead to the development of novel therapeutics for sporadic cases.</p>

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