Age-related macular degeneration (AMD) represents a significant cause of visual impairment and blindness in individuals over 65 years old. In recent years, gene therapy has emerged as a research hotspot for wet AMD, with adeno-associated virus (AAV) vectors being widely utilized due to their non-pathogenic nature, low immunogenicity, broad tissue tropism, and capacity for sustained transgene expression. Several related studies have progressed to clinical trial stages. Although challenges persist, including immunogenicity concerns, limited vector capacity, and potential long-term adverse effects, the continuous advancement of research strategies and technologies holds promise. Future developments may employ AAV delivery systems to achieve gene supplementation, gene editing, or gene silencing of angiogenesis-related signaling molecules, thereby providing novel therapeutic approaches for wet AMD.
