Hepatocellular Carcinoma: understanding the genotoxic risks of liver-targeted gene therapy using recombinant AAV vectors [ 2018 - ]

Research Grant

Researchers: Prof Ian Alexander (Principal investigator) ,  Prof Geoffrey McCaughan

Brief description Advances in gene transfer technology using an engineered virus known as AAV underpin success in the treatment of haemophila B, and offer the exciting prospect of treating many other liver diseases. While continued improvement of gene transfer efficiency is essential there is an equal need to focus on safety. We have discovered a genetic element in AAV that we believe is a key to unlocking accurate analysis of the safety of AAV-based gene transfer technology. Here we propose to turn the key.

Funding Amount $891,639.00

Funding Scheme Project Grants

Notes Standard Project Grant