Targeting endothelial cells by gene therapy.

摘要

The recent clinical successes in gene therapy are fueling renewed interest in the field. Sustained therapeutic benefits have been repeatedly observed in patients with a wide variety of severe genetic disorders and cancer. These advances mirror the continuous improvements in gene delivery technologies. Nevertheless, there is still a need to develop vectors that can specifically deliver their therapeutic gene cargo into the desired target cells while preventing unwarranted genetic modification of nontarget cells. One of the "holy grails" in gene therapy focuses on the identification of robust cell-specific "targetable" vectors that can accomplish such a feat. The specific delivery of genes into endothelial cells has been particularly challenging. In this issue, Abel et al have now overcome this bottleneck and demonstrate, for the first time, targeted gene delivery to endothelial cells on systemic LV injection.