CBM Seminar Series – Advancing Gene and Cell Therapy for Inherited Retinal Diseases

The College of Biomedicine was pleased to host Professor Dong Hyun Jo from the Department of Anatomy and Cell Biology, College of Medicine, Seoul National University, as a distinguished speaker in the CBM Seminar Series on 16 March 2026. Professor Jo is a physician-scientist specializing in ophthalmology and regenerative medicine. His research focuses on developing therapeutic strategies for inherited retinal diseases and ocular tumors by integrating patient-derived induced pluripotent stem cells (iPSCs), gene editing, and organoid technologies.

In his talk entitled "Advancing Gene and Cell Therapy for Inherited Retinal Diseases", Professor Jo addressed the significant clinical challenge of inherited retinal diseases (IRDs), which are a major cause of blindness characterized by genetic variants affecting retinal functions. He discussed how recent advances in genome editing and stem cell technologies have opened new possibilities for targeted therapeutics.

Professor Jo presented his team's translational research, illustrating the integration of gene editing and patient-derived iPSC platforms for the treatment of IRDs. He detailed their use of base and prime editing strategies to correct pathogenic variants in preclinical models, aiming to achieve functional rescue. Furthermore, he shared ongoing work on generating patient-specific iPSC-derived retinal organoid systems to accurately model disease mechanisms. The seminar also covered their efforts in developing iPSC-based cell therapy approaches, with the ultimate goal of establishing clinically applicable differentiation pipelines.

The seminar stimulated active discussion on the challenges and opportunities in translating gene- and cell-based therapies into clinical practice, underscoring the promise of precision medicine in treating currently untreatable retinal disorders.