Year 1

In the first year of this award we have developed optimized gene editing tools to allow us to make very specific changes to certain genes in the body that we hypothesize will provide protection against HIV-1 infection. We started doing this work in model cell line systems and have now moved to show that the same tools will also work in the hematopoietic stem cells that give rise to all of the cells of the blood and immune system. At the same time we are looking to find ways to deliver these tools directly in vivo to those same cells where they reside in the bone marrow, by modifying naturally occurring viruses or ‘vectors’. We have made progress towards developing new vectors that have this delivery capability.