A platform to deliver silencing RNAs using exosomes

在过去的十年里，科学家们已经发现了能够特异性和有效地抑制蛋白质的表达或功能的药物，包括抗体和沉默RNA。沉默的RNA可以深刻地抑制任何基因的表达，而抗体可以选择性地结合特定版本的蛋白质，比如那些错误排列并聚集在神经退化中的蛋白质。使用这两种药物治疗许多疾病的一个根本障碍是，它们在需要的组织中积累并穿过细胞膜进入其许多靶点所在的细胞的能力。例如，沉默RNA是改变生命的肝病新疗法，但不能用于其他组织，因为目前的递送工具效率太低，毒性太大。 最近，我们体内的细胞被发现会释放被称为外体的微小气泡，将内容物从一个细胞输送到另一个细胞。吉宾斯实验室正在开发利用外切体的这种天然能力来运送药物，如沉默RNA来治疗患者的方法。吉宾斯实验室将与一家制药公司合作，进行原则验证实验，以测试他们关于外切体的发现是否允许将其用作治疗患者的沉默RNA的平台。该项目的成功完成将对加拿大人进行生物技术方面的就业培训，并使这家制药公司能够进一步开发这些有希望的药物输送工具，对加拿大经济和加拿大人的健康有潜在的好处。此外，吉宾斯博士的目标是利用这些发现在加拿大成立一家生物技术公司，为加拿大带来进一步的经济利益。

In the past decade, scientists have discovered drugs that can specifically and potently inhibit the expression or function of proteins including antibodies and silencing RNAs. Silencing RNAs can profoundly inhibit expression of any gene, while antibodies can selectively bind specific versions of a protein, like those that misfiled and aggregate in neurodegeneration. A fundamental roadblock in the use of these two types of drugs for many diseases is there ability to accumulate in the tissues where they are required and cross the cell membrane to enter into cells where many of their targets are located. For example, silencing RNAs are life-changing new treatments for liver diseases, but cannot be used in other tissues because the current delivery vehicles are too inefficient and toxic. Recently, cells in our bodies were discovered to release tiny bubbles called exosomes that deliver contents from one cell into another. The Gibbings lab is developing methods to harness this natural ability of exosomes to deliver drugs like silencing RNAs to treat patients. Working with a pharmaceutical company the Gibbings lab will perform proof-of-principle experiments to test whether their discoveries about exosomes allow them to be used as a platform for delivery of silencing RNAs to treat patients. Successful completion of this project will train Canadians for employment in biotechnology and enable further development of these promising drug delivery vehicles by the pharmaceutical company with potential benefits to the Canadian economy and the health of Canadians. In addition, Dr. Gibbings aims to use these discoveries to launch a biotechnology company based in Canada which will provide further economic benefit to Canada.