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: $ 17.97万
• 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: 2019
• 资助国家: 加拿大
• 起止时间: 2019-01-01 至 2020-12-31
• 项目状态: 已结题
• 来源: https://www.nserc-crsng.gc.ca/ase-oro/Details-Detailles_eng.asp?id=694668
• 关键词: Selective; laser; mediated; gene; therapy

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.

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，要么通过当前的疗法只能暂时改善。干扰缺陷基因或补充缺陷基因的新兴解决方案已显示出希望。然而，目前选择性针对眼睛患病细胞的治疗方式尚存在空白，这阻碍了有效的基于基因疗法的治疗的发展。当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。用于角膜和视网膜细胞的安全、精确和选择性的基因传递系统将为解决重大公共卫生问题的疾病开辟新的治疗途径。拟议的研究项目旨在使用激光和 FDA 批准的金纳米颗粒开发一种针对角膜和视网膜的靶向非病毒基因传递治疗方法。该项目将由多学科研究人员团队执行，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。我们的团队开发了利用金纳米粒子以激光介导的基因递送到细胞的专业技术，效率高且毒性极低。基于已提交的专利以及细胞、组织和活体动物中令人兴奋的初步数据，我们将把我们的技术专业知识扩展到患病的眼睛，旨在提供一种新的、安全的、选择性的方法和系统来治疗加拿大导致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、激光技术专家 AxisPhotonics 和专有眼部相机诊断系统的所有者 Optina 一起执行。两者都将参与我们技术的商业化。