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Delivery system for Dualase and testing of potential for gene therapy in partnership with Specific Biologics

Dualase 输送系统以及与 Specific Biologics 合作测试基因治疗潜力

基本信息

批准号：
560748-2020
负责人：
Sheardown, Heather
金额：
$ 2.19万
依托单位：
McMaster University
依托单位国家：
加拿大
项目类别：
Alliance Grants
财政年份：
2020
资助国家：
加拿大
起止时间：
2020-01-01 至 2021-12-31
项目状态：
已结题

来源：
https://www.nserc-crsng.gc.ca/ase-oro/Details-Detailles_eng.asp?id=707190
关键词：
Delivery system Dualase testing potential

项目摘要

Specific Biologics Inc., a Toronto based start up, has a novel gene editing technique that they have validated for various research applications and are currently developing for use in the treatment of cystic fibrosis. The technique has greater specificity and fewer off target effects than currently available technologies. Based on the considerable interest in the use of gene therapy in ocular applications, SBI is interested in examining the potential of this protein based technology for its potential in the eye which is currently widely touted as a potential gene therapy target. In partnership with the Sheardown lab from McMaster University, which brings expertise in ophthalmic materials and drug delivery, the project will develop a method of delivering the protein based gene editor to the ocular tissue based on previously established ocular delivery systems and will examine the potential of the system to alter the genome of ocular cells in vitro and in vivo. Specifically the work will look at the specificity of the gene editing and the level of off target effects. Ultimately, this work will allow SBI to assess the ophthalmic potential of their system and to optimize the system for the treatment of a variety conditions and for research applications in the eye.

总部位于多伦多的初创公司Specific Biologics Inc.拥有一种新的基因编辑技术，他们已经在各种研究应用中进行了验证，目前正在开发用于治疗囊性纤维化的技术。与现有技术相比，该技术具有更高的特异性和更少的脱靶效应。基于基因治疗在眼部应用方面的巨大兴趣，SBI有兴趣研究这种基于蛋白质的技术在眼睛中的潜力，目前被广泛吹捧为潜在的基因治疗靶点。该项目与麦克马斯特大学的Sheardown实验室合作，该实验室带来了眼科材料和药物输送方面的专业知识，该项目将开发一种将基于蛋白质的基因编辑器输送到眼部组织的方法，该方法基于先前建立的眼部输送系统，并将检查该系统在体外和体内改变眼部细胞基因组的潜力。具体来说，这项工作将着眼于基因编辑的特异性和脱靶效应的水平。最终，这项工作将使SBI能够评估其系统的眼科潜力，并优化该系统，以治疗各种疾病并用于眼睛的研究应用。