Joost van Haasteren

In the Gene Medicine research group, I investigate the opportunities for lifelong expression of therapeutic genes to correct genetic liver diseases. Recent gene therapy clinical trials have shown that the duration of expression of therapeutic genes (which ideally would be lifelong) leaves much to be desired, causing the therapeutic effect to be only temporary.

I am investigating two possible solutions to circumvent this issue. On the one hand, I am trying to avert the antibody response that the body raises to fight the viruses used for gene therapy to make sure that those antibodies can no longer prevent the viruses from infecting the targeted liver cells. The second part of my project concerns the possibility to have a single gene therapy dose exerting a lifelong effect by integrating the therapeutic gene directly into a precise spot in the genome using a novel form of genome editing.

Before joining the University of Oxford, I did a master's degree in Cell and Gene Therapy at University College London during which I worked in the lab of Prof Stephen Hart on CRISPR/Cas9 mediated knock-down of ENaC in Cystic Fibrosis. I have completed my bachelor's degree at Maastricht University.