Grant: Proof of Principle Program - Phase I

In 2006 the Nobel Prize in Medicine was awarded for the discovery of a new type of drug called silencing RNAs. Early research showed that these drugs could be readily designed to silence virtually any gene in a very specific and powerful manner. This suggested that silencing RNAs could be used to treat virtually any disease. 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. We aim to utilize the body's natural vesicles for transporting silencing RNAs to deliver therapeutic silencing RNAs. With this funding, we will test our novel technology for packaging silencing RNAs into exosomes and test where silencing RNAs delivered by exosomes are active in the body. With this knowledge we can begin to use exosomes to deliver silencing RNAs and treat diseases associated with this tissue. In the long-term we 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.