Adeno-associated viruses for delivery of KH902 and uses thereof

Aspects of the present disclosure relate to compositions and methods for expressing an anti-vascular endothelial cell growth factor (VEGF) agent in a cell or subject. In some embodiments, the disclosure provides rAAVs comprising a capsid protein (e.g., an AAV2 variant, an AAV2/3 hybrid variant, an AAV8 variant, etc.) and a transgene encoding an anti-VEGF agent (e.g., KH902) and one or more regulatory sequences. In some embodiments, the compositions described herein may be used to treat a subject having a disease associated with angiogenesis or aberrant VEGF activity/signaling. 本公开的多个方面涉及用于在细胞或受试者中表达抗血管内皮细胞生长因子(VEGF)剂的组合物和方法。在一些实施方案中，本公开提供了包含衣壳蛋白(例如，AAV2变体、AAV2/3杂合变体、AAV8变体等)和编码抗VEGF剂(例如，KH902)的转基因以及一个或多个调控序列的rAAV。在一些实施方案中，本文所述的组合物可用于治疗患有与血管生成或异常VEGF活性/信号传导相关的疾病的受试者。