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

ABSTRACT: Hydrodynamics-based gene transfer has been successfully employed for in vivo gene delivery to the liver of small animals by tail vein injection and of large animals using a computer-assisted and image-guided protocol. In an effort to develop a hydrodynamic gene delivery procedure clinically applicable for gene therapy, we have evaluated the safety and effectiveness of a lobe-specific hydrodynamic delivery procedure for hepatic gene delivery in baboons. Reporter plasmid was used to assess the gene delivery efficiency of the lobe-specific hydrodynamic gene delivery, and plasmid-carrying human factor IX gene was used to examine the pattern of long-term gene expression. The results demonstrated liver lobe-specific gene delivery, therapeutic levels of human factor IX gene expression last

SUBMITTER: Kamimura K 

PROVIDER: S-EPMC10280096 | biostudies-literature | 2023 Jun

REPOSITORIES: biostudies-literature