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（眼前的前部）或视网膜（眼睛后部的图像加工电池）的眼部疾病是视力丧失的主要原因，并影响了数百万个单个环保的范围。这些疾病是无法治愈的，或者仅通过电流行动而瞬时改善。干扰有缺陷基因或补充缺乏的新兴解决方案表现出了希望。但是，在治疗方式中有一个目前的空隙，可以选择性地将患病的细胞在眼睛上，这阻碍了有效的基于幼体疗法的疗法的发展。当前的几种基因治疗方法要求使用病毒传递基因，但基于病毒的系统却呈现了许多有毒的副作用。 ASAFE，针对角膜和视网膜细胞的精确和选择性基因输送系统将开放治疗途径，以解决与公共卫生有关的主要关注的疾病。该计划的研究项目旨在使用激光和FDA批准的金纳米颗粒开发针对角膜和视网膜的靶向和非病毒基因递送治疗。该项目将由具有互补专业知识，纳米技术，光学，角膜和视网膜细胞生物学的互补专业知识的研究人员组成。我们的团队在激光介导的基因输送到高效率和极低的毒性金纳米颗粒中已经开发了止血症。在细胞，组织和活体动物中提交的专利和令人兴奋的初步数据的基础上，我们将使用Theaim将技术专长扩展到患病的眼睛，以提供一种新的，安全，选择性的方法和系统，以治疗加拿大的两种重要疾病：与年龄相关的黄斑变性和角膜内皮症。该项目将与我们的知识转移用户，Axisphotonics，Laser Technologies专家和Optina（专有眼球诊断系统的所有者）一起执行。两者都将参与我们技术的商业化。