年龄相关性黄斑变性（AMD）是由多因素共同诱发的视网膜黄斑区改变，基因治疗可以显著改善AMD患者临床症状，减轻长期玻璃体内治疗相关的治疗负担，提高临床适应性。尽管已取得多项成果，但基因疗法仍处于起步阶段，多数药物的临床试验仍处于研究阶段。通过整理近10年以来AMD的相关靶点治疗产品的药理研究与临床研究进展，并根据不同靶点或新载体进行分类，结合已取得的临床试验结果，评估基因治疗AMD的可行性与安全性，以期对于AMD相关靶点、治疗技术创新与载体开发提供参考。

Age-related macular degeneration (AMD), a multifactorial macular alteration of the retina. In AMD gene therapy research, gene therapy has been found to significantly improve patients' clinical symptoms, reduce the treatment burden associated with long-term intravitreal therapy, and improve clinical adaptability. Despite several achievements, current gene therapies are still in their infancy, and clinical trials of most drugs are still in the research stage. This article reviews the pharmacological research progress of AMD-related target therapy products in the past 10 years, classifies them according to different targets or new vectors, and evaluates the feasibility and safety of gene therapy AMD based on the results of clinical trials obtained, so as to provide a reference for AMD-related targets, therapeutic technology innovation and vector development.

江苏省新药一站式高效非临床评价公共服务平台建设（BM2021002）