The etiology of many diseases is not well known. To achieve a better understanding of its causes, we will utilize human pluripotent stem cell (hPSC)-derived organoid differentiation platforms, CRISPR gene perturbations, and single cell and spatial technologies. By equipped with these cutting-edge techologies, we can dissect the molecular mechanism behind of our diseases in-depth which will help us to develop the personalized treatment for patients.
Cardiac Team (Daeun Gong, Mikyoung Shim): Identification of unknown roles of epigenetic regulators during heart development using hPSC-derived cardiac organoids and CRISPR genetic screening with single cell and spatial transcriptomics.
(Related Funding: MUSCAT: Establishment and optimization of muscle cell atlas for therapeutic applications on skeletal and cardiac diseases)
Pancreas Team (Mikyoung Shim, Chaeyeon Shin, Romee van der Linden, Juhui Kim, YoonHee Kim):
1) To identify the environmental factors, such as medications, which can induce diabetes, using small molecules screening human pluripotent stem cell-derived pancreatic differentiation.
2) Generation of pancreatic beta cells from human induced pluripotent stem cells via CRISPR genome editing for cell therapy of diabetic patients.
(Related Funding: Ex-vivo gene editing for mitochondrial diabetes treatment with iPSCs by advanced base editing and prime editing)
Gene Therapy Team (Chaeyeon Shin, Juhui Kim): Development of genetic therapy strategies for rare genetic disease patients using human pluripotent stem cells or patient-derived pancreatic organoids.
(Related Funding: Development of an ultrasound sensitive guide RNA nanoparticle-microbubble complex gene editing delivery system for the prevention of hereditary pancreatitis)
Immune Team (Danbi Kang, Chaeyeon Shin, Jungwon Choi) : Using pluripotent stem cells as a resource for immune cell therapy has been a huge attention; however, the differentiation to generate functional immune cells from pluripotent stem cells has not been successful due to several molecular bottlenecks. To establish more efficient differentiation platform, we will use CRISPR genetic screening to elucidate the molecular regulation during in vitro differentiation and identify the obstacles to fix.
(Related Funding: Development of CAR-T cell therapy derived from human pluripotent stem cells)
RS-2023-00223069 MUSCAT: Establishment and optimization of muscle cell atlas for therapeutic applications on skeletal and cardiac diseases (2023-04-01~2027-12-31, Total 617,500,000 won)
RS-2023-00261247 Development of an ultrasound sensitive guide RNA nanoparticle-microbubble complex gene editing delivery system for the prevention of hereditary pancreatitis (2023-07-01~2027-12-31, Total 675,500,000 won)
RS-2024-00332601 Korean Fund for Regenerative Medicine: Ex-vivo gene editing for mitochondrial diabetes treatment with iPSCs by advanced base editing and prime editing (2024-04-01~2028-12-31, Total 712,500,000 won)
Yuhan Innovation Program Development of CAR-T cell therapy derived from human pluripotent stem cells (2024-09-01~2025-08-31, Total 100,000,000 won)