area of retina (red) lacking oxygen (green)

Gene therapy for other diseases

Neovascular disease
In several conditions, blindness is caused by the abnormal growth of blood vessels in the eye. These diseases include diabetes, macular degeneration and retinopathy of prematurity. Treatments that are currently available for these conditions are often ineffective and can cause serious side effects. Research to understand the cells and signalling molecules involved, however, has led to the development of new improved treatments that are safer and more effective. Using gene therapy we can target these new molecular medicines inside the eye in sustained way by a single injection. We have developed several candidate treatments in the lab that can control the growth of abnormal blood vessels. We are currently evaluating their efficacy and safety with a view to clinical trials in the future.

Auto-immune uveitis
The human immune system is designed to recognise, attack and destroy invading microorganisms. The immune system consists of a complex network of chemicals that either strengthen or weaken the attack, trying to find a balance between the destruction of the microorganisms and the collateral damage done to the surrounding tissue. In auto-immune diseases such as arthritis or MS, the immune system erroneously recognises a part of the body and starts attacking it, resulting in damage to that tissue. Posterior uveitis is an auto-immune disease in which the immune system damages the cells in the retina and, if left untreated, it will result in severe loss of vision. As we know which molecules can steer the immune system to a less aggressive action, we can design viral vectors that contain the genes for these molecules.

Currently we are performing animal experiments in which we are testing viral vectors containing the genes for various molecules that weaken the immune responses. In this way we try to determine which genes or combination of genes is most suitable for the amelioration of auto-immune uveitis.

This page last modified 18 December, 2012 by xxx