Advancing Precision Ophthalmology: Human Retinal Organoids as Platforms for Disease Modeling and Personalized Therapy

Human-induced pluripotent stem cell (hiPSC)-derived retinal organoids (ROs) have emerged as a transformative 3D in vitro model that closely recapitulates human retinogenesis and the layered cellular architecture of the native retina. This review highlights their profound utility as a precision platform for ophthalmic research. ROs, particularly those derived from patients with inherited retinal diseases (IRDs), provide an effective model for investigating disease pathogenesis, while retaining the patient’s genetic background. This specificity is crucial for the modeling of monogenic disorders like Retinitis Pigmentosa (RP) and Leber Congenital Amaurosis (LCA) and complex conditions like Age-Related Macular Degeneration (AMD), Diabetic Retinopathy (DR), and battle-related ocular trauma in the battlefield. Furthermore, ROs serve as a scalable and high-throughput tool for drug screening and toxicology testing, accelerating the identification of therapeutic compounds. Finally, they represent a renewable source for generating purified, functional retinal cells (e.g., photoreceptors and retinal ganglion cells) for precision cell therapies aimed at retinal repair. While challenges remain in achieving true vascularization and long-term maturation, RO technology is poised to rapidly advance personalized medicine and curative strategies for blinding retinal disorders.