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

Specific Biologics Inc.一家位于多伦多的初创公司，拥有一种新的基因编辑技术，他们已经验证了各种研究应用，目前正在开发用于治疗囊性纤维化。该技术比现有技术具有更高的特异性和更少的脱靶效应。基于在眼部应用中使用基因治疗的相当大的兴趣，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。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递送方面的专业知识，该项目将开发一种基于先前建立的眼部递送系统将基于蛋白质的基因编辑器递送到眼部组织的方法，并将研究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，这项工作将研究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，并优化系统，用于治疗各种疾病和眼睛的研究应用。

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.