Grant: Collaborative Health Research Projects

Retinal degenerative diseases are the leading causes of vision loss and affect millions of individuals in Canada. Thesediseases are either incurable or can be only moderately ameliorated with current therapies. As an emerging solution,delivering therapeutic genes into impaired cells shows promising results in proof of concept studies, and several clinical trialsare undergoing. However, in most cases, gene delivery is based on viral vectors, whose safety is controversial. Viral vectorspresent disadvantages, such as size limitation, high immunogenicity and potential mutations of transduced cells. Hence, thelack of efficient, non-toxic and persistent gene delivery strategies undermine the full potential of gene transfer therapiesagainst retinal degenerative diseases. The proposed research program aims to develop a non-viral ocular gene deliverytechnique using ultrafast lasers amplified by plasmonic gold nanoparticles. Our method has been proven able to delivernaked genes into cells with high efficiency and very low cytotoxicity. The proposed project includes optimising the technologyfor retinal neurons, refining tissue selectivity with biofunctionalized nanoparticles specifically targeting diseased cells, anddetermining the effectiveness of laser gene delivery in vivo by using mouse models of retinal neurodegeneration. Given theextensive use of lasers in current ophthalmic practice, our technology may be seamlessly inserted to existing clinical setupsfor treatment of retinal degenerative diseases. This project will be performed by a multidisciplinary team of researchers withhighly complementary expertise in femtosecond lasers, biophotonics, ocular optics and retinal cell biology in closecollaboration with two user organisations: Optina Diagnostics and the Foundation for Fighting Blindness.