Review

Gene therapy for inborn errors of liver metabolism: progress towards clinical applications

Nicola Brunetti-Pierri

Department of Molecular and Human Genetics, Baylor College of Medicine, Houston, TX, USA

author email corresponding author email

Italian Journal of Pediatrics 2008, 34:2doi:10.1186/1824-7288-34-2

Published:	18 November 2008

Abstract

The treatment for inborn errors of liver metabolism is based on dietary, drug, and cell therapies (orthotopic liver transplantation). However, significant morbidity and mortality still remain, and alternative strategies are needed. Gene replacement therapy has the potential of providing a definitive cure for patients with these diseases. Significant progress has been made in the pre-clinical arena and achievement of efficacy in different animal models has been reported using multiple gene transfer technologies. This article summarizes the gene transfer strategies being investigated, the pre-clinical data, and the available early clinical results for inborn errors of liver metabolism.