Functional restoration of OTC deficient primary human hepatocytes in a xenograft model using an AAV vector uniquely configured for impending clinical trial use. [ 2015 - 2016 ]

Research Grant

Researchers: Prof Ian Alexander (Principal investigator) ,  Prof Paul Gissen

Brief description The aim of this project is to acquire preclinical data which will underpin an international gene therapy trial for severe ornithine transcarbamylase (OTC) deficiency, the most prevalent urea cycle defect in infants and children. In most severe cases, liver transplantation is required for long term survival. We, with colleagues at Stanford University, have recently developed a novel gene therapy tool for optimal targeting of human liver cells which will be tested in a humanised mouse model.

Funding Amount $AUD 235,525.52

Funding Scheme Project Grants

Notes Standard Project Grant