Retinal degenerative disease (RD) is the main cause of visual impairment. Traditional models have limitations and treatment options are limited. Retinal organoids (RO), as an emerging 3D culture technology, can differentiate stem cells in vitro to form multi-layered tissues that highly simulate the structure and function of the retina, bringing a breakthrough approach to RD research. At present, this technology has been successfully applied to the model construction of diseases such as retinitis pigmentosa, retinoblastoma and Leber congenital amaurosis. Based on patient-specific RO, the study revealed pathological mechanisms such as ciliary defects and protein aggregation caused by mutations, and verified the potential of gene editing, adeno-associated virus therapy and targeted drugs. Meanwhile, RO has demonstrated efficient and reliable evaluation capabilities in drug screening. Despite still facing challenges such as long cycles, high costs, and a lack of vascular and immune cells, future research will focus on optimizing the culture system, constructing vascularized and immunized organoids, and promoting clinical transformation. At present, RO transplantation has demonstrated safety and repair potential in animals and preliminary clinical trials, laying a foundation for its application in retinal regeneration and precision medicine.
