Zonal expression and persistence of rAAV vectors within the hepatic lobule: Towards gene therapy for metabolic disease. [ 2008 - 2013 ]

Research Grant

Researchers: Dr Allison Dane (Principal investigator)

Brief description In its most severe form the urea cycle disorder OTC deficiency has a high risk of death. Current therapy is liver transplantation which has significant shortcomings including lack of donor organs. Therefore, a need to provide a safer and more effective alternative treatment exists. Liver directed gene therapy with adeno-associated virus (AAV) can potentially provide this treatment. This project aims to optimise AAV2-8 vectors for liver gene therapy in the treatment of metabolic liver disease.

Funding Amount $AUD 84,173.64

Funding Scheme NHMRC Postgraduate Scholarships

Notes Dora Lush Biomedical Postgraduate Scholarship