Research Overview

Our group focuses on rare intractable diseases, using patient-specific iPS cells to elucidate pathogenic mechanisms and develop much-needed therapies. Many rare and intractable diseases that develop during childhood have unknown disease mechanisms or no established treatment. Since these diseases often require lifelong medical attention, innovations in diagnostic and therapeutic methods are highly desirable.

For diseases that cannot be readily modeled using experimental animals or cultured cell lines, iPS cell-based disease modeling represents a powerful tool for understanding their pathophysiology. Our research mainly focuses on immune diseases (immunodeficiency and autoinflammatory syndromes), hematological diseases (hematopoietic and leukemia-like disorders), and neuromuscular diseases. We are also searching for drugs to suppress specific disease phenotypes after reproducing them in vitro. We wish to continue our endeavor to contribute to the diagnosis and treatment of as many patients as possible.