A platform to deliver silencing RNAs using exosomes

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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.