Capsid variants and uses thereof

Aspects of the present disclosure relate to compositions and methods for delivering a transgene (e.g., a transgene encoding one or more gene products) to a target cell. The present disclosure is based, in part, on adeno-associated virus (AAV) capsid protein variants characterized by tropism to certain cell types (e.g., neurons, muscle cells, bone cells, cardiac cells, etc.). In some embodiments, recombinant AAVs (rAAVs) comprising the capsid protein variants (e.g., AAVv66, SEQ ID NO: 1) are more efficiently packaged than rAAVs having certain wild type AAV capsid proteins. The present disclosure also describes methods of delivering rAAVs comprising the AAV capsid protein variants. 本公开的多个方面涉及用于将转基因(例如，编码一种或多种基因产物的转基因)递送至靶细胞的组合物和方法。本公开部分基于特征在于对某些细胞类型(例如，神经元、肌肉细胞、骨细胞、心脏细胞等)的嗜性的腺相关病毒(AAV)衣壳蛋白变体。在一些实施方案中，包含所述衣壳蛋白变体(例如，AAVv66，SEQ ID NO:1)的重组AAV(rAAV)比具有某些野生型AAV衣壳蛋白的rAAV更有效地包装。本公开还描述了递送包含所述AAV衣壳蛋白变体的rAAV的方法。