The present provides methods and compositions that enable effective delivery of nucleic acids to desired cells, including to a solid organ such as a mammalian heart. The methods and compositions enable effective gene transfer and subsequent expression to a majority of cells throughout a solid organ such as the heart. Methods and compositions of the invention preferably provide enhanced vascular permeability that enables increased gene transfer to targeted cells, but without significant degradation or injury to endothelial cell layers.
本文提供了一种方法和组合物,可以有效地将
核酸传递到所需的细胞,包括哺乳动物心脏等固体器官。这些方法和组合物可以实现有效的
基因转移,并随后在固体器官(如心脏)的大多数细胞中表达。本发明的方法和组合物最好提供增强的血管通透性,从而使
基因转移到靶向细胞的数量增加,但不会对内皮细胞层造成显著的降解或损伤。