FUTURE

Our project this year is the first phase of a two-year investigation in collaboration with Dr. Kagimoto from Healios Japan KK. This year, we focused on A1AT deficiency and used CRISPR, gene-editing technology, to address the point mutation in the SERPINA1 gene. We first developed a system within E. Coli cells to show proof-of-concept and confirm whether we were able to accurately address the mutation. Next, we intend to test for use in mammalian cells and observe its development and effectiveness in mice. We plan on advancing our system by working with induced pluripotent stem cells. iPS cells are generated directly from adult cells and thus eliminate the ethical problems associated with embryonic stem cells. We hope to incorporate this into a liver organ bud that could be used as an alternative treatment for diseases caused by genetic mutations in the liver. An organ bud, which is 3D and transplantable, is a condensed tissue mass of the early stages of organ formation resulting from the coculture of iPS cells and multiple progenitors. By using an organ bud, those affected by this type of disease would have access to a one-time treatment instead of requiring regular injections. It would also the need for a liver transplant. This would be more cost-effective and practical for patients. Additionally, research conducted by Takanori Takebe of Yokohama City University has shown that liver organ buds can be used to create a vascularized and functional 3D liver organ, massively broadening the horizons in the field regenerative medicine. Once we further our understanding of this new technology, we plan on expanding our project to work with other diseases using induced pluripotent stem cells.