Research Overview

Our group conducts research on pathological analysis and development of therapy using iPS cells specific for rare intractable diseases. Many rare and intractable diseases that develop in childhood have unknown mechanisms of onset or have no established treatment. Since these diseases can cause lifelong suffering, innovations in diagnostic and therapeutic methods are desired.

For diseases which are not suitable for research using animal models or cultured cell lines, it is useful to apply disease-specific iPS cells to understand their pathophysiology. Our research mainly focuses on immune diseases (immunodeficiency and autoinflammatory syndromes), hematological diseases (hematopoietic disorders and leukemia-like disorders), and neuromuscular diseases. We are also searching for drugs to suppress the specific phenotype of the disease after reproducing it in vitro. We would like to continue our research to contribute to the diagnosis and treatment of as many patients as possible.