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AAV-based Fusion Protein Targeting Human VEGFR-2 Domains Shows Promise in Treating Neovascular- Associated Retinal Diseases in Mice

Abstract

Yin Cao

Neovascular-associated retinal diseases, including age-related macular degeneration and diabetic retinopathy, are leading causes of vision loss worldwide. Vascular Endothelial Growth Factor Receptor 2 (VEGFR-2) plays a critical role in angiogenesis, making it an attractive therapeutic target for these diseases. In this study, we developed an Adeno-Associated Virus (AAV)-based fusion protein specifically targeting human VEGFR-2 domains to evaluate its efficacy in treating neovascular-associated retinal diseases in mice. The AAV-based fusion protein was designed to consist of a Single-Chain Antibody Fragment (scFv) derived from a high-affinity VEGFR-2 antibody, fused with a potent anti-angiogenic peptide. The scFv component enabled specific binding to VEGFR-2, while the anti-angiogenic peptide aimed to inhibit downstream signaling pathways involved in angiogenesis. The fusion protein was packaged into an AAV vector for efficient delivery to retinal cells.

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