Gene therapy is a therapeutic strategy that aims to employ nucleic acids as drugs for the transient or permanent treatment of inherited or acquired pathologies. Based on the type of vector employed for the gene transfer, gene therapy can be classified as viral gene therapy and nonviral gene therapy. Nonviral gene therapy is less efficient but safer than viral gene therapy. Hydrodynamic naked DNA transfer has shown great translational potential, achieving therapeutic levels of a human protein in the murine model. The translational process of the procedure has already been performed. Different radiologic and surgical approaches permitted pressurizing the liver in vivo by excluding its vascularization partially or totally. These approaches mediated a tissue rate of human alpha-1-antitrypsin protein translation (100–1000 copies per cell) close to those obtained with the mouse gold standard model in a safe mode that could be translated to human settings.
Part of the book: Liver Research and Clinical Management