Application progress of gene therapy targeting vascular endothelial growth factor in ocular fundus vascular diseases

The pathogenesis of ocular vascular diseases mainly involves the excessive expression of vascular endothelial growth factor (VEGF), leading to abnormal proliferation of retinal neovascularization and/or increased vascular permeability, such as neovascular age-related macular degeneration and diabetic retinopathy, which seriously threaten patients’ vision. The application of anti-VEGF drugs has revolutionized the treatment methods for ocular vascular diseases. However, significant challenges remain unresolved in clinical practice, including the burden of frequent injections, high costs, and treatment resistance. With the rapid development of gene therapy technology, new paths for the clinical treatment of ocular vascular diseases have been opened up and have shown significant advantages. This article introduces the research and application progress of gene therapy targeting VEGF in ocular vascular diseases. It elaborates on the mechanism of VEGF in promoting abnormal vascular growth and increasing vascular permeability in ocular vascular diseases, and details the current VEGF-based gene therapy strategies, such as using gene silencing technology to target and reduce VEGF expression, and using gene editing techniques, such as clustered regularly interspaced short palindromic repeats-associated protein to modify VEGF-related genes. At the same time, it discusses the application status and challenges of these treatment methods in preclinical and clinical trials, such as the safety and effectiveness of vectors and immune responses. It also looks forward to the future development prospects of VEGF-based gene therapy in ocular vascular diseases, aiming to provide a reference for in-depth research and clinical application in this field.