Grant: Project Grant

Neurological conditions affect millions of Canadians. Unfortunately, such conditions are particularly challenging to treat due to difficulty in accessing the brain and the lack of specialized tools to enable treatments. New tools for manipulation of brain tissues are desperately needed. One approach with enormous potential for enabling novel and potentially life-changing treatments is genetic drugs - the delivery of a genetic sequence encoding one or more biologically active proteins that act to resolve complex diseases processes. Lipid nanoparticles (LNP) are the current best delivery system for genetic cargo, such as mRNA, with an excellent safety profile as evidenced by the billions of administered shots of the COVID-19 mRNA vaccines. Here we propose to develop LNPs for delivery of mRNA into brain tissues. Experiments in our labs have demonstrated that mRNA can be effectively delivered to white matter in the brain after intraventricular delivery. Our objective is to further develop this LNP-mRNA formulation as a platform delivery system for genetic drugs in the brain. For proof-of-concept, we have chosen stroke as a disease indication, and aim to show the function of our system by synthetic expression of a growth factor known to be important for post-stroke recovery. We aim to both develop a platform technology for genetic drug delivery in the brain and demonstrate its functional potential in a relevant disease model.