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

Niigata, Japan - Professor Kamimura's research group at Niigata University has successfully utilized a groundbreaking liver lobe-specific hydrodynamic delivery technique on primates, specifically baboons. This marks the first instance of such a procedure being performed on this animal species. According to Prof. Kamimura, the introduction of a therapeutic gene for human hemophilia through plasmid delivery resulted in sustained expression of the human factor IX gene at therapeutic levels for a remarkable duration of 200 days.

The research outcomes not only established the effectiveness of repetitive hydrodynamic gene delivery to the same liver lobes but also demonstrated the procedure's specificity to the liver without affecting other organs in the baboons undergoing gene therapy. Furthermore, apart from a transient rise in liver enzyme levels immediately following the injection, no significant adverse events were observed in the animals throughout the study period.

Based on these findings, Prof. Kamimura concluded that this procedure holds great promise for clinical applications, offering a safe and effective therapeutic approach for a wide range of challenging diseases.