Stem Cell-Based Study for Drug Discovery for PolG disease
Lay Abstract
The project aims to establish a stem cell-based platform for drug discovery and therapeutic development in patients with PolG-related diseases. PolG mutations are associated with various neurological phenotypes, and the mechanisms underlying the disease progression are not fully understood.
To overcome the limitations of animal models and the lack of accessibility to neural tissue, the project utilizes induced pluripotent stem cells (iPSCs) derived from patients. These iPSCs can be differentiated into different types of neuronal cells, including neural stem cells (NSCs) and dopaminergic (DA) neurons, which exhibit mitochondrial dysfunction similar to that observed in patient postmortem brains.
This project aims to establish a stem cell-based neural system and 3D brain organoid platform using human cells for drug discovery. The specific focus is on identifying mitochondria-targeting therapies for patients with PolG mutations, a genetic disorder affecting mitochondrial function.
By leveraging stem cell technology and utilizing 2D and 3D models, this project aims to provide insights into the pathophysiology of PolG-related diseases and identify potential therapies for further clinical investigation. The ultimate goal is to discover mitochondria-targeting drugs that can be repurposed for PolG treatment, offering new avenues for therapeutic intervention and improving the lives of patients affected by these debilitating diseases.
Institution
Faculty of Medicine, University of Bergen, Norway
University of Bergen
Faculty of Medicine
Postbox 7804
NO-5020 BERGEN, NORWAY
Principle Investigator
Kristina Xiao Liang, Ph.D
Cell Biology and Mitochondrial Biology at the University of Bergen
Dr. Kristina Xiao Liang is a Principal Investigator and Senior Researcher specializing in Cell Biology and Mitochondrial Biology at the University of Bergen, located in Norway. With 10 years of experience, her long-term focus on stem cell and mitochondrial research makes her a pioneering authority on the use of human induced pluripotent stem cell (iPSC) models to explore mitochondrial diseases. In her lab, she explores the complex world of human stem cell/iPSC model systems to understand diseases caused by mutations in mitochondrial DNA polymerase (PolG). Additionally, she has spearheaded the development of drug screening platforms aimed at discovering new therapeutic strategies. Her extensive work has been published in journals such as EMBO Molecular Medicine, Cellular and Molecular Life Sciences, and Frontiers in Cellular and Developmental Biology etc. She has a Ph.D. from the University of Bergen. She then enriched her knowledge with sufficient postdoctoral training at the Department of Clinical Medicine, also at the University of Bergen. Her rich educational background and professional achievements demonstrate her unwavering commitment to advancing the field of stem cell research in mitochondrial disease research.