Selective laser-mediated gene therapy for ocular diseases

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Ocular diseases affecting the cornea (front part of the eye) or the retina (image processing**cells in the back of the eye) are leading causes of vision loss and affect millions of individuals**worldwide. These diseases are either incurable or only transiently improved with current**therapies. Emerging solutions that interfere with defective genes or supplement deficient ones**have shown promise. However, there is a current void in treatment modalities that selectively**target diseased cells on the eye, which has hindered the development of effective genetherapy-**based treatments. Several current gene therapy approaches call for the use of**viruses to deliver genes, yet virus-based systems present a number of toxic side-effects. A**safe, precise and selective gene delivery system for corneal and retinal cells would open**novel therapeutic avenues to address diseases that are of major public health concern. The**proposed research project aims to develop a targeted and non-viral gene delivery treatment**for the cornea and the retina using lasers and FDA approved gold nanoparticles. This project**will be performed by a multidisciplinary team of researchers with complementary expertise in**lasers, nanotechnology, optics, corneal and retinal cell biology. Our team has developed an**expertise in laser-mediated gene delivery into cells with high efficiency and very low toxicity**using gold nanoparticles. Building on a filed patent and exciting preliminary data in cells,**tissues and in live animals, we will expand our technical expertise to diseased eyes, with the**aim to offer a new, safe, selective method and system to treat two important diseases leading**to blindness in Canada: age-related macular degeneration and corneal endothelial**dystrophies. The project will be performed with our Knowledge Transfer Users, Axis**Photonics, experts in laser technologies and Optina, owner of a proprietary eye camera**diagnostic system. Both will participate in the commercialisation of our technique.