Research progress in adeno-associated virus vectors for gene therapy of wet age-related macular degeneration
10.3760/cma.j.cn511434-20250226-00075
- VernacularTitle:腺相关病毒载体在渗出型年龄相关性黄斑变性基因治疗中的研究进展
- Author:
Fei LI
1
;
Xinrong XU
1
Author Information
1. 南京中医药大学附属医院眼科, 南京 210029
- Publication Type:Journal Article
- Keywords:
Age-related macular degeneration;
Gene therapy;
Adeno-associated virus;
Viral vector;
Review
- From:
Chinese Journal of Ocular Fundus Diseases
2025;41(9):734-738
- CountryChina
- Language:Chinese
-
Abstract:
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.
