In vivo gene transfer and phenotype correction of normal and urea-cycle deficient primary human hepatocytes in chimeric mouse-human livers: Towards gene therapy for metabolic liver disease

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1008021

Funding Status
Closed

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Funded Activity Summary

Genetic liver disease imposes a major health and economic burden. Existing medical treatments are frequently inadequate, often necessitating liver transplantation which carries its own limitations and risks. Using a gene therapy approach we have achieved life-long cure of mice with OTC-deficiency, a condition with a high risk of disability and death in affected infants. This application focuses on translating this success in mice through to human therapy.

Funded Activity Details

Start Date: 01-01-2011

End Date: 01-01-2013

Funding Scheme: NHMRC Project Grants

Funding Amount: $493,747.00

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

gene therapy | genetic disorders | liver | liver disease | metabolic disorders

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