Selective laser-mediated gene therapy for ocular diseases

影响角膜（眼睛前部）或视网膜（眼睛后部的图像处理细胞）的眼部疾病是导致视力丧失的主要原因，影响着全世界数百万人。这些疾病要么无法治愈，要么用目前的疗法只能短暂改善。干扰缺陷基因或补充缺陷基因的新解决方案显示出了希望。然而，目前在选择性靶向眼部病变细胞的治疗方式上存在空白，这阻碍了有效的基于基因疗法的治疗方法的发展。目前的一些基因治疗方法要求使用病毒来传递基因，然而基于病毒的系统存在许多毒副作用。一种安全、精确和选择性的角膜和视网膜细胞基因传递系统将为解决主要公共卫生关注的疾病开辟新的治疗途径。拟议的研究项目旨在开发一种针对角膜和视网膜的靶向和非病毒基因传递治疗，使用激光和FDA批准的金纳米颗粒。该项目将由一个多学科研究团队进行，他们在激光、纳米技术、光学、角膜和视网膜细胞生物学方面具有互补的专业知识。我们的团队已经开发出一种利用金纳米粒子高效、低毒性的激光介导基因传递到细胞中的专业技术。基于已申请的专利和令人兴奋的细胞、组织和活体动物的初步数据，我们将把我们的技术专长扩展到患病的眼睛，目的是提供一种新的、安全的、选择性的方法和系统来治疗加拿大两种导致失明的重要疾病：年龄相关性黄斑变性和角膜内皮营养不良。该项目将与我们的知识转移用户AxisPhotonics，激光技术专家和Optina一起执行，Optina拥有专有的眼睛相机诊断系统。双方都将参与我们技术的商业化。

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.