Development of a novel hybrid rAAV/transposon gene delivery system for life-long correction of metabolic liver disease in infants and children

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1065053

Funding Status
Closed

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Funded Activity Summary

The immense potential of gene therapy for the treatment of genetic liver disease has been confirmed by recent success in a clinical trial for Haemophilia in adult males, and therapeutic benefit in other adult trials is imminent using the same technology. In the young, however, ongoing growth of the liver causes the therapeutic benefit to be short-lived. To address this problem we are developing a powerful new hybrid technology capable of conferring life-long benefit on infants and children.

Funded Activity Details

Start Date: 01-01-2014

End Date: 01-01-2016

Funding Scheme: Project Grants

Funding Amount: $505,897.00

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 | transduction | transposons | virus

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