Liver sinusoidal endothelial cells as possible vehicles for gene therapy: a comparison between plasmid-based and lentiviral gene transfer techniques.

Unlabelled: Liver sinusoidal endothelial cells (LSECs) constitute an attractive target for gene therapy of several liver and systemic diseases. However, there are few reports showing an efficient plasmid-based or viral methodology to deliver recombinant genes into these cells. In the present study, the authors evaluated in vitro gene transfer efficiency of standard plasmid-based techniques (i.e., electroporation, lipofection, and calcium phosphate) and lentiviral-mediated gene transduction into primary murine LSECs, using reporter genes. The results show that electroporation is the most effective in vitro plasmid-gene transfer method to deliver GFP into LSECs (31%), as compared with lipofection and calcium phosphate transfection (6% and 4%, respectively). However, lentiviral transduction resulted in higher, efficient, and stable gene transfer (70%) as compared with plasmid-based techniques.

Conclusions: The highly efficient gene expression obtained by lentiviral transduction and electroporation shows that these methodologies are highly reliable systems for gene transfer into LSECs.