A platform to deliver silencing RNAs using exosomes

使用外泌体传递沉默 RNA 的平台

• 批准号: 539153-2019
• 负责人: Gibbings, Derrick
• 金额: $ 5.11万
• 依托单位: University of Ottawa
• 依托单位国家: 加拿大
• 项目类别: Collaborative Research and Development 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=699551
• 关键词: platform; deliver; silencing; RNAs; using

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.

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包括抗体和沉默rna在内的蛋白质表达或功能的药物。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一种蛋白质的特定版本，比如那些在神经变性中错误排列和聚集的蛋白质。在许多疾病中使用这两种药物的一个基本障碍是，它们能够在需要它们的组织中积累，并穿过细胞膜进入它们的许多靶标所在的细胞。例如，沉默rna是改变肝脏疾病生活的新疗法，但不能用于其他组织，因为目前的运载工具效率太低，毒性也太大。