Seminar announcement: Dr. Hidetoshi Sakurai - "Development of Novel Therapeutic Options for Muscular Dystrophy Utilizing iPS Cell Technology"

Establishment of induced pluripotent stem (iPS) cells enables us to proceed with therapeutic development research into various intractable diseases because of its infinite proliferation potential and pluripotency. We have been investigating the development of new therapies to treat muscular diseases by two distinct approaches using iPS cell technology.

One is cell transplantation therapy by the iPSC-derived muscle stem cells (iMuSCs) having high skeletal muscle regenerating potential. We have developed a differentiation method to mimic the developmental steps and succeeded in inducing iMuSCs applicable to cell transplantation therapy. Then we have been pursuing our research for clinical application of iMuSC therapy toward Duchenne muscular dystrophy (DMD) while developing surface markers to purify iMuSC, or xeno-free coating material for efficient iMuSC induction. We also revealed functional improvement by dystrophin restoration by cell transplantation.

Another strategy is drug discovery using patient-derived iPS cells through in vitro disease modeling. We have also successfully induced skeletal myocytes from hiPSCs in highly reproducible and efficient manner with short differentiation period mediated by MyoD overexpression. In modeling DMD, we demonstrated that continuous electric stimulation promoted maturation of myotubes followed by recapitulation of fatigue-like phenotype in vitro. We are now trying to generate 3D muscle organoids which can represent more mature phenotypes enough for recapitulation of disease phenotypes in many other muscular diseases.

Short CV
Title: Dr. (M.D., Ph.D.)
Position: Associate Professor
Institution: Center for iPS Cell Research and Application (CiRA), Kyoto University

Degrees

- 1998 – M.D., School of Medicine, Nagoya University
- 2005 – Ph.D., Graduate School of Medicine, Nagoya University

Professional Experience

- October 2015–present – Principal Investigator (Associate Professor), CiRA, Kyoto University
- November 2009–September 2015 – Principal Investigator (Lecturer), CiRA, Kyoto University
- June 2008–October 2009 – Postdoctoral Fellow, CiRA, Kyoto University
- November 2005–May 2008 – Postdoctoral Fellow, Department of Immunology, Nagoya University School of Medicine
- April 2001–October 2005 – Ph.D. research at the Stem Cell Group, Center for Developmental Biology, RIKEN, Kobe, in Shin-ichi Nishikawa’s laboratory, while enrolled in the Graduate School of Medicine, Nagoya University