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 processingcells in the back of the eye) are leading causes of vision loss and affect millions of individualsworldwide. These diseases are either incurable or only transiently improved with currenttherapies. Emerging solutions that interfere with defective genes or supplement deficient oneshave shown promise. However, there is a current void in treatment modalities that selectivelytarget diseased cells on the eye, which has hindered the development of effective genetherapy-based treatments. Several current gene therapy approaches call for the use ofviruses to deliver genes, yet virus-based systems present a number of toxic side-effects. Asafe, precise and selective gene delivery system for corneal and retinal cells would opennovel therapeutic avenues to address diseases that are of major public health concern. Theproposed research project aims to develop a targeted and non-viral gene delivery treatmentfor the cornea and the retina using lasers and FDA approved gold nanoparticles. This projectwill be performed by a multidisciplinary team of researchers with complementary expertise inlasers, nanotechnology, optics, corneal and retinal cell biology. Our team has developed anexpertise in laser-mediated gene delivery into cells with high efficiency and very low toxicityusing 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 theaim to offer a new, safe, selective method and system to treat two important diseases leadingto blindness in Canada: age-related macular degeneration and corneal endothelialdystrophies. The project will be performed with our Knowledge Transfer Users, AxisPhotonics, experts in laser technologies and Optina, owner of a proprietary eye cameradiagnostic system. Both will participate in the commercialisation of our technique.