Liver lobe-specific hydrodynamic gene delivery to baboons: A preclinical trial for hemophilia gene therapy

Niigata, Japan – The research group of Professor Kamimura in Niigata University have applied the novel, liver lobe-specific hydrodynamic delivery procedure to primates (baboons) for the first time. “Delivery of a plasmid that expresses a therapeutic gene for human hemophilia achieved therapeutic levels of human factor IX gene expression lasting for 200 days after the delivery of a plasmid”, says Prof. Kamimura. In addition, the results demonstrated the efficacy of repeated hydrodynamic gene delivery into the same liver lobes. Furthermore, no plasmid was introduced into organs other than the livers of the baboons treated with gene therapy, and other than a transient increase in blood concentration of liver enzymes right after the injection, no significant adverse events were observed in animals during the study period. Prof. Kamimura concluded that this procedure can be safely applied to the clinic and will show the therapeutic effect in various intractable diseases.