Selective laser-mediated gene therapy for ocular diseases

影响角膜(眼睛的前部)或视网膜(眼睛后部的图像处理细胞)的眼病是导致视力丧失的主要原因，影响着全球数百万人**。这些疾病要么是无法治愈的，要么只能通过目前的**疗法暂时得到改善。干扰缺陷基因或补充缺陷基因的新兴解决方案**已经显示出希望。然而，目前选择性地针对眼睛上的病变细胞的治疗方式存在空白，这阻碍了有效的基于基因疗法的治疗的发展。目前的几种基因治疗方法要求使用**病毒来传递基因，但基于病毒的系统存在许多毒副作用。一种**安全、准确和选择性的角膜和视网膜细胞基因输送系统将开辟**新的治疗途径，以解决引起重大公共卫生问题的疾病。这项拟议的研究项目旨在利用激光和FDA批准的金纳米颗粒，开发一种针对角膜和视网膜的靶向和非病毒基因传递疗法。这个项目**将由一个多学科的研究团队实施，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**使用金纳米颗粒以高效和非常低的毒性**将激光介导的基因输送到细胞中的**专业知识。基于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、有选择性的方法和系统来治疗在加拿大导致**失明的两种重要疾病：老年性黄斑变性和角膜内皮营养不良。该项目将与我们的知识传授用户、Axis**Photonics、激光技术专家和拥有专有眼部相机**诊断系统的Optina一起执行。这两家公司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 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.