色素上皮细胞衍生因子(p igm ent ep ithelium-derived factor,PEDF)是一种相对分子质量为50 000的分泌蛋白,属于丝氨酸超家族的一员。近年来研究发现PEDF具有多种生物学功效,包括抗新生血管,神经营养及神经保护功能。PEDF是最有效的内源性眼部新生血管抑制物。PEDF对于治疗血管增生性疾病及视网膜变性性疾病具有广阔的前景,基因转移为眼内组织治疗药物的持续传递提供了有效途径。就近年来关于PEDF基因治疗视网膜及脉络膜疾病的研究进展给以综述。 PEDF is a secreted protein with a relative molecular mass of 50,000, which belongs to the serine superfamily. In recent years, studies have found that PEDF has a variety of biological effects, including antiangiogenic, neurotrophic and neuroprotective functions. PEDF is the most effective endogenous ocular neovascularization inhibitor. PEDF has broad prospects for the treatment of vascular proliferative diseases and retinal degenerative diseases and gene transfer provides an effective way for the sustained delivery of intraocular tissue therapeutic drugs. In recent years, the research progress of PEDF gene therapy for retinal and choroidal diseases are reviewed.