|Hidetoshi Sakurai M.D.,Ph.D.
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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
In our strategy to establish cell transplantation treatment for muscular dystrophy, we try to differentiate iPS cells into muscle progenitor cells, and the muscle progenitor cells differentiate into satellite cells, skeletal muscle stem cells, in vivo following engraftment into dystrophic model animals. We believe that the proliferation of the skeletal muscle stem cells derived from iPS cells will contribute to the regeneration of host impaired muscle, and that normal muscle fiber will increase enough to yield therapeutic effect.
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.