The ultimate goal of our research is to exploit new advances in stem cell biology and gene editing technology to develop a deeper understanding for the basis of neurological diseases including Alzheimer's, Parkinson’s and Neuropsychiatric diseases, and ultimately to develop new therapies for these diseases.
Stem cell technology has created new opportunities in understanding human disease, drug discovery, and regenerative medicine. We are particularly interested in the idea that cell reprogramming has the great potential to provide specific cell types for regenerative medicine applications, so we are aiming at replacing tissues that have been lost or damaged due to degenerative diseases and injury with cell reprogramming technology.
Also, we recently develop a human organoid-based drug discovery platform for neurodegenerative diseases. Organoids are hPSC-derived three-dimensional in vitro culture systems that recapitulate the developmental processes and organization of the developing human organ. These ‘mini-organs’ provide a physiologically relevant in vitro 3D model for the study of development and disease processes that are unique to the human organ system. Particularly, we are interested in generating patient specific brain orgnaoids which can be used for in vitro drug screening and target validation as new clinically relevant disease models with the ultimate goal of developing personalized therapeutic interventions in complex multifactorial human neurological diseases.
In parallel, we have recently developed a CRISPR/Cas9 gene targeting based novel therapeutics. The therapeutic gene targeting in patients provides an exciting opportunity to not only explore the mechanisms of the diseases but also to evaluate the potential of this strategy for clinical application in human diseases.
To address these goals, we utilize the various biotechnolgoy, transgenic mouse system, with biochemical and computational biology approaches.