Regenerative Medicine Project
Integration of iPS cell and genome editing technologies to cure genetic disorders
Yuichiro Miyaoka has been the leader of the Regenerative Medicine Project since 2016.
He received his Ph.D. from the Institute of Molecular and Cellular Biosciences, the University of Tokyo under the supervision of Dr. Atsushi Miyajima in 2009. After receiving his Ph.D., he worked as a staff scientist in the Dr. Atsushi Miyajima’s lab from 2009 to 2011. Then, he did his postdoctoral training in the Bruce Conklin’s lab at Gladstone Institutes, USA from 2011 to 2015, where he developed the first digital PCR-based method to detect genome editing outcomes. He applied this method to isolate genome-edited cells without antibiotic selection. His current interest is to apply genome editing in human iPS (induced pluripotent stem) cells to cure genetic disorders by disease modeling, cell transplantation therapy, and direct genetic manipulation in patients’ cells. For these therapeutic applications, genome editing should be precise. Therefore, he also aims to improve the accuracy and predictability of genome editing.
Backgrounds
TiPS cells will be useful in developing treatments for a wide range of diseases. However, difficulties in editing the genomes of iPS cells have been a major hurdle in this process. It is now becoming possible to overcome this hurdle by using novel genome editing technologies.
By combining iPS cell and genome editing technologies, our group aims to develop new models for genetic disorders. We will use these models to elucidate the pathogenesis of these diseases. We will also design new cell-based therapies that will involve correcting genetic mutations in iPS cells by genome editing.
Objectives
- We will introduce pathogenic mutations into iPS cells from healthy patients to model genetic diseases. We will use these modified iPS cells to study the pathogenesis of these disorders
- We will develop methods to correct pathogenic mutations in iPS cells from patients with genetic disorders. We will test these cells for recovery of cellular functions, and use them to develop cell transplantation therapies
Members
Project Leader Yuichiro Miyaoka
- Tomoko Kato