Dept. of Clinical Application
Hidetoshi Sakurai (Associate Professor)
Hidetoshi Sakurai M.D., Ph.D.
Our laboratory aims to establish remedies for refractory muscle diseases, specifically, muscular dystrophy. There are two ways for treatment we hope to attain. One is cell transplantation and the other is drug development. Regarding the former, we use progenitor cells differentiated from iPS cells as a source for cell transplantation, and evaluate therapeutic effects with disease-model animals. Regarding the latter, we use iPS cells established from somatic cells taken from patients as a tool for drug discovery, by making study on the model construction in order to reproduce disease states in vitro.
Development of cell transplantation
As cell transplant therapy for muscular dystrophy, we envisage regenerating and repairing damaged muscle fibers by transplanting skeletal muscle stem cells generated from our iPS Cell Stock for Regenerative Medicine. The aim is to achieve successful engraftment following in vivo differentiation into satellite cells, a type of skeletal muscle stem cell; in vivo proliferation of these satellite cells will then contribute to repeated regeneration and further therapeutic effect by increasing the amount of healthy muscle fiber.To treat a muscular disease which is caused by the defect of interstitial protein, we aim to achieve therapeutic effect by supplementing the insufficient protein in the interstitial tissue through transplant of mesenchymal stromal cells generated from the iPS Cell Stock.
Construction of disease models
iPS cells have advantage to be created from anybody as long as he/she has skin cells. Taking this advantage, we conduct joint research with pediatricians and neurologists for the purpose of constructing disease models with the use of iPS cells derived from muscular dystrophy patients. We make research on how to induce highly efficient differentiation of adult skeletal muscles. We also investigate the possibility to reproduce the disease state of muscular dystrophy by culturing differentiation-induced skeletal muscle with the physical stress culturing system.
Meanwhile, through joint research, mainly with pharmaceutical manufacturers, we have developed a screening system for use with diseases that we have successfully modeled and are seeking compounds that may contain the seed of a drug. Using a different approach, we are studying whether genome editing to repair genetic mutations can relieve disease. If this approach proves effective, we will proceed with research aimed at developing genome-editing therapies.