Hepatic gene therapy: efficient retroviral-mediated gene transfer into rat hepatocytes in vivo.

"Hepatic Gene Therapy: Efficient Retroviral Mediated Gene Transfer into Rat Hepatocytes

Therapeutic levels of functional human factor X in rats after retroviral-mediated hepatic gene therapy.

Factor X deficiency results in a rare but serious bleeding disorder that might be treated by expressing a normal factor X gene in patients. We generated an amphotropic retroviral vector with the human FX cDNA and delivered it to rat hepatocytes in vivo during liver regeneration. The human alpha1-antitrypsin promoter was chosen to direct expression because it was the most efficient of several te...

Hepatocyte growth factor induces hepatocyte proliferation in vivo and allows for efficient retroviral-mediated gene transfer in mice.

Recombinant retroviral vectors are an attractive means of transferring genes into the liver because they integrate into the host cell genome and result in permanent gene expression. However, efficient in vivo gene transfer is limited by the requirement of active cell division for integration. Traditional approaches to induce liver proliferation have the disadvantage of inducing hepatocellular i...

Portal branch occlusion safely facilitates in vivo retroviral vector transduction of rat liver.

Hepatic gene therapy might correct the clinical manifestations of several genetic disorders in patients. Although retroviral vectors with a strong liver-specific promoter can result in stable and therapeutic levels of expression of genes from the liver, application of these techniques in humans is limited by the need to perform one or more invasive procedures to achieve ex vivo or in vivo trans...

Human foetal hepatocytes: isolation, characterization, and transplantation.

Hepatocyte transplantation has become an alternative to orthotopic liver transplantation for the treatment of liver metabolic diseases. However, there is an increasing lack of donor organs and isolated mature hepatocytes are difficult to manipulate and cannot be expanded in vitro. It is therefore necessary to find alternative sources of hepatocytes, and different approaches to evaluate the ther...