Gene therapy gains pace

Optometrist Jasleen Jolly and the team of researchers based at Oxford Eye Hospital are making great strides in gene therapy, offering hope for many patients for which their condition currently has no treatment.

The gene therapy treatment that the Oxford team is working on is to treat choroidermia, a rare inherited disorder that causes progressive vision loss. With positive results to date, Ms Jolly confirmed that they have now begun looking at this treatment for RPGR mutation too. 

Speaking about how gene therapy works, Ms Jolly explained: “Gene therapy is for patients with inherited retinal degeneration. They have a defective gene, which doesn’t produce the protein that they need, and that is what leads to cell is the retina dying. 

“What we are doing is replacing the gene using a gene embed in the virus that is delivered to the back of the eye and then that gene is taken up, starts procuring the protein and therefore you stop any further vision loss as the eye is working properly.”  

Passionate about the potential that gene therapy has for people with inherited retinal diseases, Ms Jolly believes that this work will open up a huge number of options for a big group of patients.  “For inherited disease, we have never had an option to restore vision before, but now a lot of technologies are being developed in the lab to help protect from further vision loss, or even in future restore vision,” she highlighted.