Successful liver-directed gene delivery by ERCP-guided hydrodynamic injection (with videos).

ABSTRACT:

Background and aims

A simple, safe, targeted, and efficient in vivo DNA delivery system is necessary for clinical-grade liver-targeted gene therapy in humans. Intravascular hydrodynamic gene delivery has been investigated in large animal models, but translation to humans has been hampered by its technical challenges, invasiveness, and potential for significant cardiovascular adverse events. We posited that intrabiliary delivery of DNA plasmids via ERCP-guided hydrodynamic injection could overcome these obstacles.

Methods

Twelve pigs (40-50 kg) were divided into 3 groups (4 per group) and survived 21, 30, or 60 days. ERCP was performed by inflating a balloon catheter in the common hepatic duct and creating a closed space between it and the liver parenchyma. Last, a solution c

SUBMITTER: Kumbhari V 

PROVIDER: S-EPMC6249687 | biostudies-literature | 2018 Oct

REPOSITORIES: biostudies-literature