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 [ 2011 - 2013 ]

Research Grant

Researchers: Prof Ian Alexander (Principal investigator) ,  A/Pr Filip Braet ,  A/Pr Kevin Carpenter ,  Prof John Christodoulou ,  Prof Philip Kuchel

Brief description 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.

Funding Amount $AUD 493,747.86

Funding Scheme NHMRC Project Grants

Notes Standard Project Grant