Drug delivery: silencing RNAs using exosomes


Competition: EXPLORE Program 2014
Funding: $300,000 / 2 years
Beginning: June 2015

Early research has shown that a new type of molecule called silencing RNAs could be readily designed to silence or inhibit virtually any gene in a very specific and powerful manner. This in turns eliminates the expression of the protein encoded by this gene. Because most diseases could benefit from shutting-down the action of a specific protein, it is suggested that silencing RNAs could be used to treat virtually any diseases. Unfortunately, a roadblock has prevented use of silencing RNAs to treat most diseases: the ability to deliver these drugs into the tissues and organs where they are needed. The body makes its own equivalent of silencing RNAs, and uses tiny vesicles called exosomes to transport these between cells. The project aims to utilize these natural vesicles to deliver therapeutic silencing RNAs. Derrick Gibbings will test his novel technology for packaging silencing RNAs into exosomes and test where silencing RNAs delivered by exosomes are active in the body. With this knowledge he can begin to use exosomes to deliver silencing RNAs and treat diseases associated with this tissue. In the long-term he will modify exosomes to target other tissues and enable treatment of further diseases. This would enable the treatment of many previously untreatable diseases with silencing RNAs.

Derrick Gibbings

University of Ottawa