Intrabiliary infusion of naked DNA vectors targets periportal hepatocytes in mice

A retrograde approach for liver gene transfer

“…In a study published in Molecular Therapy – Methods & Clinical Development , Deplazes and colleagues rejuvenated the hydrodynamic intrabiliary injection originally developed by Wolff and applied it to mouse models of inherited metabolic diseases. 5 Delivery via hydrodynamic retrograde intrabiliary injection (HRII) of DNA minicircles ( Figure 1 ) encoding the OTC gene in spf-ash mice, a model of ornithine transcarbamylase (OTC) deficiency, did not result in sufficient therapeutic efficacy, and further optimization is needed. Nevertheless, the method is clinically attractive because it could be performed through endoscopic retrograde cholangiopancreatography (ERCP), a method that has been in clinical practice for many years and was previously tested by the authors in a large-animal model.…”