Retinal degenerative diseases are a group of blinding ocular disorders characterized by progressive and irreversible loss of retinal neural cells. They mainly include age-related macular degeneration and retinitis pigmentosa. At present, there are no curative clinical therapies capable of reversing the disease course. Patients have poor long-term visual prognosis, imposing a heavy public-health burden on families and society. Mesenchymal stem cell(MSC)-derived exosomes, as a novel cell-free therapeutic vector, have emerged as a research hotspot due to their low immunogenicity, no tumorigenic risk, and excellent tissue barrier penetration ability. This review systematically summarizes the core mechanisms of MSC-derived exosomes in regulating immune inflammation, inhibiting apoptosis, and promoting retinal neuroprotection and structural/functional recovery via miRNA and tsRNA, compares the efficacy differences and existing controversies among exosomes from different sources, and outlines the advances in intraocular delivery technologies and engineering modification strategies. Combined with preclinical and clinical data, the current bottlenecks and safety evidence for clinical translation were objectively described. Meanwhile, in view of the heterogeneity among existing studies, this review proposes future directions including standardized preparation and the establishment of efficacy evaluation systems, so as to provide theoretical references for the development of novel therapeutic strategies for retinal degenerative diseases.

江西省卫生健康委员会科技计划项目(No.202210872)