The Program for Intractable Diseases Research utilizing Disease-specific iPS cells
Center for iPSC production
Production of disease-specific iPS cells from somatic cells deposited by nation-wide research/clinical institutes
| First Year | FY2012 |
|---|---|
| Title | Fundamental research to promote establishment of disease-specific induced pluripotent stem (iPS) cells |
| Principal Investigator | Shinya Yamanaka (Director and Professor, Center for iPS Cell Research and Application, Kyoto University) |
| Summary | The ability to establish iPS cells as pluripotent stem cells derived from specific individuals, including patients, constitutes extremely important progress, and analysis of refractory disease pathology, drug creation, and development of treatment methods are expected to be achieved using disease-specific iPS cells established from patients. On the other hand, great progress has been achieved with genetic modification of human embryonic stem cells and iPS cells, in connection with advances in reprogramming technology, and it is expected that the era of genetic modification of cells of these types, and disease and pathology analysis on this basis, is about to dawn. The objectives of this center are to establish various disease-specific iPS cells; to prepare human iPS cells with modifications of genes responsible for diseases; to place such cells in public cell banks; and to establish a Japanese research base for disease analysis, drug development, etc. |
Collaborative research
Clinical research and drug discovery of intractable and rare diseases
| First Year | FY2012 |
|---|---|
| Title | Development of in vitro models with high-quality differentiated cells and tissues aiming at the pathogenesis and therapy for refractory diseases of the nervous and visual systems |
| Principal Investigator | Haruhisa Inoue (Professor, Center for iPS Cell Research and Application, Kyoto University) |
| Subsidiary institution | RIKEN |
| Summary | This center aims to apply technologies that have been generated by previous development in fields such as regenerative medicine, including human induced pluripotent stem (iPS) cell establishment, differentiation, and purification technologies, so as to prepare model cells and tissues for nervous and visual diseases, followed by technology transfer to clinical researchers in the Japanese Ministry of Health, Labour and Welfare’s Refractory Disease Research Group. On these bases, the aim is to coordinate research on refractory diseases of the visual, nervous and neuroendocrine systems, the etiology and pathology of which are still unclear in many respects, and to contribute to development of important treatment methods and therapeutic agents. |
| First Year | FY2012 |
|---|---|
| Title | Research on intractable neurological diseases using disease-specific induced pluripotent stem (iPS) cell technology |
| Principal Investigator | Hideyuki Okano (Professor, School of Medicine, Keio University) |
| Subsidiary institution | University of Tokyo |
| Summary | The aims of this project are to further increase the efficiency of previously developed disease-specific human iPS cell establishment systems, and culture systems for neural cell differentiation; to accelerate research on neurodegenerative diseases through studies of neurological disease-specifc iPS cells.
It is almost impossible to perform cell biological and biochemical analysis of pathological conditions in neurodegenerative diseases because of the difficulties to access to the lesion areas in patients. However, it is hoped that disease-specific iPS cells will make it possible to replicate certain features of human disease in vitro. In this collaborative research with the Japanese Ministry of Health, Labour and Welfare’s intractable Disease Research Groups., we establish iPS cells from patients with neurological diseases and differentiate them into disease-relevant cell types. |
| First Year | FY2012 |
|---|---|
| Title | Elucidation of the pathology of hereditary myocardial diseases, and development of treatment methods, using induced pluripotent stem (iPS) cells |
| Principal Investigator | Hiroyuki Morita(Acting Principal Investigator) (Project Associate Professor, Graduate School of Medicine, The University of Tokyo) |
| Subsidiary institution | Osaka University, Keio University, National Center for Child Health and Development, Kyoto University |
| Summary | This center aims to elucidate the pathologies of, and develop treatments for, hereditary myocardial diseases, including cardiomyopathy and hereditary arrhythmia. At the present time, no appropriate treatment methods are available for hereditary myocardial diseases, and there is therefore a demand for realization of disease-specific treatments. The objectives of the present research are to prepare iPS cells and iPS-derived myocardiocytes from hereditary myocardial disease patients; to elucidate the pathologies of hereditary myocardial diseases, in collaboration with the Japanese Ministry of Health, Labor and Welfare’s Refractory Disease Research Group; to establish screening methods for rectifying abnormalities of iPS-derived myocardiocytes specific to hereditary myocardial diseases, in collaboration with pharmaceutical companies; and to identify compounds as starting points for development of therapeutic agents specific to hereditary myocardial diseases. |
| First Year | FY2012 |
|---|---|
| Title | Research on refractory musculoskeletal diseases using disease-specific induced pluripotent stem (iPS) cells |
| Principal Investigator | Junya Toguchida (Professor, Institute for Frontier Medical Sciences and Center for iPS Cell Research and Application (Deputy Director), Kyoto University) |
| Subsidiary institution | National Center of Neurology and Psychiatry |
| Summary | This research group is to be involved with pathology research and new drug screening in musculoskeletal fields. There are numerous hereditary diseases in the bone and cartilage, and skeletal muscle fields. At present, for most of these diseases not only are no radical therapies available, but it is not possible to inhibit progression or alleviate symptoms. As these diseases are systemic, they can hardly be targets of regenerative medicine using cells, and there is therefore a pressing need for development of novel treatment methods. In the present research, iPS cell strains are to be established from somatic cells collected, with collaboration with the Japanese Ministry of Health, Labour and Welfare’s Refractory Disease Research Group, from patients with rare, refractory, musculoskeletal diseases. Quality evaluation is then to be carried out using differentiation-induction techniques developed previously, and, by making this available for elucidation of pathology and research on new drug development, the aim is to progress with development of revolutionary treatment methods. |
| First Year | FY2012 |
|---|---|
| Title | Establishment of disease-specific induced pluripotent stem (iPS) cells derived from refractory blood and immunological disease patients, and development of new medical treatment |
| Principal Investigator | Tatsutoshi Nakahata (Professor and Deputy Director of Center for iPS Cell Research and Application, Kyoto University) |
| Subsidiary institution | University of Tokyo |
| Summary | The aims of this center are to elucidate refractory blood and immunological diseases, and to develop new treatment methods, using disease-specific iPS cells. Most radical treatments for blood diseases depend on transplantation of hematopoietic stem cells. With many diseases, there is a demand for treatment methods that are more specific and less invasive. The present research, which involves collaboration with the Refractory Disease Research Group of the Japanese Ministry of Health, Labour and Welfare, will establish iPS cells from somatic cells collected from refractory blood and immunological disease patients, after which disease specific cells will be induced by differentiation of iPS cells. The Refractory Disease Research Group will then use the iPS cells and differentiation-induced cells to elucidate the pathology of the disease, so as to develop new drugs and medical treatment methods for refractory blood and immunological diseases. |
Research Center Network for Realization of Regenerative Medicine
Japan Agency for Medical Research and Development(AMED)
Regenerative Medicine Division,
Department of Strategy Promotion
1-7-1 Otemachi, Chiyoda-ku,Tokyo, 100-0004 Japan
Phone: +81-3-6870-2220
E-Mail：saisei-ML
amed.go.jp
Regenerative Medicine Division,
Department of Strategy Promotion
1-7-1 Otemachi, Chiyoda-ku,Tokyo, 100-0004 Japan
Phone: +81-3-6870-2220
E-Mail：saisei-ML
amed.go.jp
