Application of iPS cell technologies to treat skeletal disease

Abstract: The bone has two types of cartilage, articular cartilage and growth plate cartilage. Articular cartilage covers the end of bone and provides lubrication to diarthrodial joints. Trauma or degeneration of articular cartilage causes joint pain during motion, leading to the onset of osteoarthritis. Growth plate cartilage, where the bone grows in children, and its dysfunction due to genetic mutations causes skeletal dysplasia. The conditions that compromise articular cartilage or growth plate cartilage are poorly understood, and curative drugs are not available. iPS cell technologies are being used to study these skeletal diseases. We have been developing a method in which human iPS cells are differentiated into chondrocytes, the cells that constitute cartilage. We are generating effective and safe human iPS cell-derived cartilage as regenerative medicine to treat focal articular cartilage defects. We have confirmed their safeness and efficacy using animal transplantation models. We have also generated iPS cells from patients with FGFR3 chondrodysplasia. We found that chondrocytes derived from patient-iPS cells produce abnormal cartilage formation and thus offer a unique cellular disease model. In this seminar, we will introduce our projects to treat skeletal disease.