Enhancing ocular gene therapy using glycosidic enzymes

点击翻译按钮获取中文摘要

Gene therapy shows great promise in treating human blindness due to genetic diseases affecting the retina. Healthy genes, contained in modified viruses, are delivered by injection under the retina, a thin later of tissue that converts light into signals that are sent to our brain. However, this approach can potentially damage the retina and cannot deliver the genes across the entire retina. A potentially safer and more effective approach would be to inject the gene-carrying viruses into the vitreous, a gel-like structure in front of the retina. However, to date this approach has not worked very well with only a small number of retinal cells receiving the gene. My research to date has shown that if we use enzymes in combination with the viruses we achieve much better gene delivery to the retina following injection into the vitreous. The aim of this project is to optimise the intravitreal combination of virus and enzymes to deliver genes right across the retina. I will then see if I can demonstrate recovery of retinal function in a mouse that, because of genetic alterations, has no vision. This may ultimately lead to exciting advancements in treating patients with a variety of retinal diseases.