Retinal Degeneration and Drug Discovery Laboratory

Research Focus

Inherited retinal degeneration causes irreversible vision loss, and currently, effective treatments for most patients are unavailable. Our research focuses on understanding the molecular mechanisms underlying photoreceptor death and developing new therapies for patients. Zebrafish is a powerful vertebrate system for modeling retinal disease and enabling in vivo drug discovery. Through zebrafish studies, we have defined signaling pathways regulating retinal development and photoreceptor degeneration. One central focus of our work is in vivo quantitative high-throughput drug screening (qHTS). Using an inducible zebrafish model of retinitis pigmentosa (RP), we have screened approximately 3,000 compounds and identified novel neuroprotective candidates, several of which were validated in mouse RP models. We have further expanded this screening across multiple CRISPR-based zebrafish RP models, aiming to identify gene-agnostic candidates. In addition, we use precise gene editing to generate disease-relevant models carrying human mutations for genetic and drug screening. Our goal is to translate these discoveries into therapies that slow or halt vision loss in patients.