The demand of donor livers for transplantation exceeds the supply. In an attempt to
maximize the number of potentially usable donor livers, several centers are exploring the …

Hydrodynamic gene delivery has proven to be a safe and efficient procedure for gene
transfer, able to mediate, in murine model, therapeutic levels of proteins encoded by the …

Hepatic gene therapy by delivering non-integrating therapeutic vectors in newborns remains
challenging due to the risk of dilution and loss of efficacy in the growing liver. Previously we …

Introduction Expressing exogenous genes after naked DNA delivery into hepatocytes might
achieve sustained and high expression of human proteins. Tail vein DNA injection is an …

Different diseases lead, during their advanced stages, to chronic or acute liver failure, whose
unique treatment consists in organ transplantation. The success of intervention is limited by …

The non-viral, integrating Sleeping Beauty (SB) transposon system is efficient in treating
systemic monogenic disease in mice, including hemophilia A and B caused by deficiency of …

A hemodynamic study of hydrodynamic gene delivery (HGD) from the tail vein in rodents has
inspired a mechanism and an approach to further improve the efficacy of this procedure …

Gene therapy returns to the center stage of medicine to treat patients with diseases that are
unable to be cured with the conventional therapeutic strategies. This development is due to …

Aim Liver transplantation is the only curative strategy for final stage liver diseases. Despite
the great advances achieved during the last 20 years, the recipient immune response after …

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 …