Adeno-associated viral (AAV) vectors have been increasingly used for gene therapy in preclinical and clinical studies. In liver-directed approaches the AAV vectors have emereged as the most promising gene transfer vector in terms of safety and efficacy. A few studies have found increased incidence of hepatocellular carcinomas (HCCs) in mice injected with AAV vectors as […]

CCR5 is the major co-receptor for human immunodeficiency virus (HIV). The New England Journal of Medicine recently published the results of a clinical trial investigating the safety of the infusion of autologous CD4 T-cells in which the CCR5 gene was rendered permanently dysfunctional by a zinc-finger nuclease (ZFN). Following infusion of genetically edited cells, the CD4 T-cell count increased significantly […]

The first demonstrating of a functional human organ (i.e. liver) generated from pluripotent stem cells has been recently reported in Nature. Specified hepatic cells (immature endodermal cells destined to track the hepatic cell fate), endothelial cells and mesenchymal cells self-organized into three-dimensional liver buds in vitro that were transplanted in recipient mice. The vasculature of liver bud transplants became functional […]