Development of a new therapeutic approach for retinitis pigmentosa based on the iPS cell research

2020 Fiscal Year Final Research Report

• Project/Area Number: 18K09422
• Research Category: Grant-in-Aid for Scientific Research (C)
• Allocation Type: Multi-year Fund
• Section: 一般
• Review Section: Basic Section 56060:Ophthalmology-related
• Research Institution: Keio University
• Principal Investigator: Ozawa Yoko 慶應義塾大学, 医学部(信濃町), 特任准教授 (90265885)
• Project Period (FY): 2018-04-01 – 2021-03-31
• Keywords: 網膜 / 神経保護 / iPS細胞

Outline of Final Research Achievements

Retinitis Pigmentosa (RP) is a leading cause of blindness, worldwide. There are currently no treatments in the world. We previously reported that endoplasmic reticulum (ER) stress is one of the pathogenesis of RP, using RP patient-derived induced pluripotent stem cells (Yoshida, Ozawa, Okano et al. Mol. Brain 2014). In the current study, we performed in vivo analyses using RP model mice to show the proof of concept of the new therapeutic approach for RP focusing on ER stress.

Free Research Field

医学

Academic Significance and Societal Importance of the Research Achievements

iPS細胞研究から発した新しい研究手法を用いた研究である点に大きな学術的意義があり、これまでに治療法の無い網膜色素変性に対する新規治療法の開発につながる研究である点に大きな社会的意義がある。疾患の分子メカニズムに着目した解析であり、病態に即した治療法の提案となる点が重要である。