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

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1080333

Funding Status
Closed

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Funded Activity Summary

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.

Funded Activity Details

Start Date: 01-01-2015

End Date: 01-01-2016

Funding Scheme: Project Grants

Funding Amount: $235,525.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Gene and Molecular Therapy

ANZSRC Socio-Economic Objective (SEO)

There are no SEO codes available for this funding activity

Other Keywords

child health | gene delivery | gene therapy | liver disease | metabolic disease

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