Cell Therapy and Drug Discovery Research based on Next-Generation Gene Transfer Technology / Artificial Chromosome Vectors
Chromosomes are like a "ship (vector)" that orderly carries genes, known as blueprints of living organism. Humans have as many as 20,000 genes on 46 chromosomes. We use this chromosome "ship" for a wide range of research area, from basic to applied research. The artificial chromosome is a large "empty ship (chromosome)", from which the baggages (genes) are removed (Fig. 1). Conventional vectors (such as viral vectors) are like small ships, whereas artificial chromosome is a large ship like luxury cruise ship and has a unique feature of carrying many genes. We have been developing technology for many years to create and use this luxury cruise ship. At present, this luxury cruise ship is used for the treatment of intractable diseases and drug discovery research, and it attracts a lot of attention from domestic and overseas.
Until now, we have been working on the application of Duchenne muscular dystrophy for gene and cell therapy, the development of animals carrying human drug-metabolizing enzyme genes for prediction of human drug metabolisms, and the development of fully humanized antibody-producing animals. Cells and animals, used to evaluate the therapeutic efficacy and safety of pharmaceuticals or obtain drug candidates, are called pharmaceutical development platforms. Tottori University-launched Venture "Trans Chromosomics Inc." is currently launching a pharmaceutical development platform based on artificial chromosomes.
A joint proposal by Tottori University and Tottori Prefecture was adopted in the 2016 project for Regional Science and Technology Demonstration Base Establishment Project by the Ministry of Education, Culture, Sports, Science and Technology (MEXT), only in Chugoku-Shikoku area. The " Tottori Pharmaceutical Innovation Center" was newly established inside of Chromosome Engineering Research Center, Yonago Medical School campus. For creating innovative pharmaceuticals, including antibody drugs, through joint research with academic and pharmaceutical companies, in July 2018, we have started full-scale research using the pharmaceutical development platforms we have cultivated. One of the resident companies, Trans Chromosomics Inc., recruits students graduated from School of Life Science, Faculty of Medicine, Tottori University (a training course for researchers), or natives of Tottori and Shimane Prefecture (U-Turn), and has begun to create a framework for regional revitalization cycles by locally fostering excellent human resources and sharing the results and profits for local communities.
We hope to contribute to the creation of "Contributing to Human Health" outcomes, by working with university-launched venture and pharmaceutical companies to develop pharmaceuticals through the use of artificial chromosome vectors, a distinctive technology developed at Tottori University. Furthermore, by accelerating commercialization, we will promote the employment of local human resources and create "Next Generation Pharmaceutical Innovation from Tottori" through industry-government-university collaboration.
- Uno N, Abe S, Oshimura M, Kazuki Y. Combinations of chromosome transfer and genome editing for the development of cell/animal models of human disease and humanized animal models. J Hum Genet. 2018 Feb;63(2):145-156.
Yasuhiro Kazuki, Ph.D.
Associate Professor, Department of Biomedical Science,
Institute of Regenerative Medicine and Biofunction,
Graduate School of Medical Science, Tottori University,
Chromosome Engineering Research Center (CERC)
Director, Tottori Pharmaceutical Innovation Center (TPIC)