年齡相關(guān)性黃斑變性（AMD）是由多因素共同誘發(fā)的視網(wǎng)膜黃斑區(qū)改變，基因治療可以顯著改善AMD患者臨床癥狀，減輕長期玻璃體內(nèi)治療相關(guān)的治療負擔(dān)，提高臨床適應(yīng)性。盡管已取得多項成果，但基因療法仍處于起步階段，多數(shù)藥物的臨床試驗仍處于研究階段。通過整理近10年以來AMD的相關(guān)靶點治療產(chǎn)品的藥理研究與臨床研究進展，并根據(jù)不同靶點或新載體進行分類，結(jié)合已取得的臨床試驗結(jié)果，評估基因治療AMD的可行性與安全性，以期對于AMD相關(guān)靶點、治療技術(shù)創(chuàng)新與載體開發(fā)提供參考。

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.

江蘇省新藥一站式高效非臨床評價公共服務(wù)平臺建設(shè)（BM2021002）