Selective laser-mediated gene therapy for ocular diseases

选择性激光介导的眼部疾病基因治疗

• 批准号: 523670-2018
• 负责人: Meunier, Michel
• 金额: $ 9.29万
• 依托单位: École Polytechnique de Montréal
• 依托单位国家: 加拿大
• 项目类别: Collaborative Health Research Projects
• 财政年份: 2018
• 资助国家: 加拿大
• 起止时间: 2018-01-01 至 2019-12-31
• 项目状态: 已结题
• 来源: https://www.nserc-crsng.gc.ca/ase-oro/Details-Detailles_eng.asp?id=659695
• 关键词: Selective; laser; mediated; gene; therapy

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.

影响角膜（眼睛的前部）或视网膜（眼睛后部的图像处理 ** 细胞）的眼科疾病是视力丧失的主要原因，影响着全世界数百万人 **。这些疾病要么是无法治愈的，要么只是暂时改善与目前的 ** 疗法。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，目前在选择性地靶向眼睛上的患病细胞的治疗方式中存在空白，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需要使用 ** 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、精确和有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问题开辟新的治疗途径。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，为角膜和视网膜开发一种靶向和非病毒基因递送治疗 **。该项目 ** 将由一个多学科的研究人员团队进行，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了一种 ** 使用金纳米颗粒的激光介导基因传递到细胞中的专业技术，具有高效率和非常低的毒性 **。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、有选择性的方法和系统来治疗导致加拿大失明的两种重要疾病：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、Axis**Photonics、激光技术专家和Optina（专有眼科相机 ** 诊断系统的所有者）一起执行。双方都将参与我们技术的商业化。