Treatment of genetic liver disease by homologous recombination in vivo, coupled with a pharmoco-genetic strategy for selective expansion of genetically repaired hepatocytes

Funding Activity

Website
http://purl.org/au-research/grants/nhmrc/1080330

Funding Status
Closed

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Funded Activity Summary

This project seeks to exploit recent advancements in our ability to precisely “edit” and correct mutations underlying human genetic diseases. To improve therapeutic efficiencies of the system, we will deliver the technology using highly efficient virus-based systems and apply a novel post-repair selection process to preferentially repopulate the liver with gene-repaired cells. Demonstration of the strategy in a humanised mouse model will provide important preclinical data for human applications.

Funded Activity Details

Start Date: 01-01-2015

End Date: 01-01-2018

Funding Scheme: Project Grants

Funding Amount: $920,836.00

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 delivery | gene targeting | gene therapy | liver | pharmacogenetics

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