grant

Development of a novel hybrid rAAV/transposon gene delivery system for life-long correction of metabolic liver disease in infants and children [ 2014 - 2016 ]

Also known as: Development of a novel gene delivery system for treatment of metabolic liver disease in infants and children

Research Grant

[Cite as https://purl.org/au-research/grants/nhmrc/1065053]

Researchers: Prof Ian Alexander (Principal investigator) ,  A/Pr Kevin Carpenter

Brief description 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.

Funding Amount $AUD 505,897.94

Funding Scheme Project Grants

Notes Standard Project Grant

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