Treatment of genetic liver disease by homologous recombination in vivo, coupled with a pharmoco-genetic strategy for selective expansion of genetically repaired hepatocytes [ 2015 - ]

Research Grant

Researchers: Prof Ian Alexander (Principal investigator) ,  Prof Andras Nagy ,  Prof Markus Grompe

Brief description 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.

Funding Amount $885,084.00

Funding Scheme Project Grants

Notes Standard Project Grant