Grant: CIHR Fellowship

To address the need for therapies that eliminate HIV infection, we propose development of a new method to deliver non-viral genome editing enzymes to the immune cells within the body to render them resistant to the virus. The specialized genome editing enzymes we develop will be targeted specifically to immune cells at the front lines of HIV infection, turning off genes required for HIV infectivity and eliminating the need for continued antiretroviral therapy. This method will be compatible with intravenous administration, preventing the expensive and labor-intensive ex vivo procedures currently associated with immune cell genome editing. Once this technology is developed it will be adaptable to correcting mutations associated with genetic diseases, as we will be able to replace the immune-cell targeting moiety to recognize other cells and tissues and program the genome editing enzymes to edit the gene of interest. The ability to perform genome editing of specific human cells and tissues in vivo will be transformative to the field of therapeutic genome editing, drastically improving access to genetic therapies.