NCE (Year 4)

The overall goal of this project is to use a gene therapy approach to engineer immune system stem cells with molecules that will direct mature immune cells developed from the stem cells to attack and kill cells infected with the AIDS virus (HIV). The approach chosen, if successful, will develop a way to stably engineer the anti-viral immune response, which should provide an inexhaustible source of HIV-specific immune cells that can fight the infection in the body. We proposed to design and test this strategy in the laboratory and in a humanized mouse system that closely mimics conditions in the human body, for efficacy against HIV-1 infection. To date we have largely met our scheduled milestones associated with construction of anti-viral molecules and with the testing of various stem cell preparations in the laboratory, and in humanized mice. The group is interacting effectively and we anticipate continued progress towards our goals through additional funding.