Keio University

Development of a Novel Treatment for Cerebral Contusion Combining Genome Editing Technology and iPS Cells

Publish: May 18, 2023
Public Relations Office

May 18, 2023

Keio University School of Medicine

A research group led by Professor Masahiro Toda of the Department of Neurosurgery, Keio University School of Medicine, has demonstrated that human iPS cell-derived neural stem cells (NSCs) migrate toward damaged brain tissue, and is advancing research into safe regenerative medicine for the therapeutic application of NSCs to improve brain function.

In this study, the researchers improved motor function in mouse models of cerebral contusion by using genome editing technology to integrate a suicide gene into iPS cells, inducing them into "therapeutic NSCs," and then transplanting them into the brain. Furthermore, by administering a prodrug, they were able to eliminate any undifferentiated NSCs that remained after transplantation. This makes it possible to avoid the risk of tumor formation from transplanted cells, a significant concern in regenerative medicine using iPS cells.

Therapeutic NSCs are expected to migrate to the site of injury in the brain and potentially improve impaired brain function. To realize safe regenerative medicine for cerebral contusion, preparations are currently underway to produce clinical-grade therapeutic NSCs with the aim of starting clinical trials soon.

These research findings were published in the online edition of the scientific journal STEM CELLS on April 8, 2023 (JST).

Please see below for the full press release.

Press Release (PDF)