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AdPEDF is a gene therapy that uses an adenoviral vector to deliver the human pigment epithelium-derived factor (PEDF) gene into ocular tissues. PEDF is a naturally occurring antiangiogenic protein that inhibits factors such as fibroblast growth factor, platelet-derived growth factor, interleukin-8, and vascular endothelial growth factor. The therapy aims to treat neovascular (wet) age-related macular degeneration (AMD) by reducing abnormal blood vessel growth in the retina. After intravitreal injection of the viral vector carrying the PEDF gene, retinal cells produce PEDF protein locally for several months. Early clinical studies have shown that AdPEDF was well tolerated and may provide antiangiogenic activity in patients with advanced neovascular AMD[6][8].
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