Suzuki, Keiichiro

Graduate School of Engineering Science, Osaka UniversityProfessor*Profile is at the time of the award.

2018Inamori Research GrantsBiology & Life sciences

Inamori Research GrantsRecipients

Research topics
Development of in vivo genome-editing method for therapeutic purpose
Keyword
Summary
Targeted genome editing via engineered nucleases is revolutionizing biomedical research and holds tremendous potential for clinical applications. Despite rapid advances in the field, in vivo targeted transgene integration is still infeasible because current tools are inefficient, especially for non-dividing cells, which compose most adult tissues. This poses a tremendous barrier for uncovering fundamental biological principles and developing treatments for a broad range of devastating genetic disorders. Based on CRISPR-Cas9, I previously devised a homology-independent targeted integration (HITI) strategy, which allows for robust DNA knock-in in non-dividing cells in vitro and, more importantly, in vivo (e.g., neurons and skeletal muscles of postnatal mammals). As a proof of concept of its therapeutic potential, I previously demonstrated the efficacy of HITI in improving visual responses using a rat model of blindness, retinitis pigmentosa. In this project, I propose to improve the HITI method and establish more versatile gene-editing tool for basic research and genome-editing therapies.

Message from recipient

I would like to express my sincere gratitude for having my research project selected for the 2018 Inamori Research Grants Program. Having set up a new lab after returning home in August 2017 from the U.S., I have found this grant highly useful and encouraging. I will make efficient use of this research support as I work on basic research that always takes novel approaches.

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Biology & Life sciences