Zonal expression and persistence of rAAV vectors within the hepatic lobule: Towards gene therapy for metabolic disease.

Website
http://purl.org/au-research/grants/nhmrc/477110

Funding Status
Closed

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Funded Activity Summary

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.

Funded Activity Details

Start Date: 2008

End Date: 2013

Funding Scheme: NHMRC Postgraduate Scholarships

Funding Amount: $84,173.00

Funder: National Health and Medical Research Council

Research Topics

ANZSRC Field of Research (FoR)

Gene Therapy

ANZSRC Socio-Economic Objective (SEO)

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No related researchers have been discovered for grant Zonal expression and persistence of rAAV vectors within the hepatic lobule: Towards gene therapy for metabolic disease..

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No related organisations have been discovered for grant Zonal expression and persistence of rAAV vectors within the hepatic lobule: Towards gene therapy for metabolic disease..