A gene therapy technique developed using mice that ‘upgrades’ the immune system to resist the HIV virus has been successfully trialled in humans. Some people are born with a very rare mutation that protects them from HIV by changing the structure of their immune cells. The therapy involves extracting a sample of HIV patients’ immune cells, culturing them in vitro, and editing the cells’ DNA to give them the same protective mutation. The small-scale human trial was designed to test whether the treatment was safe and effective in humans, but further study will be required to determine whether it can replace drug treatments in the long run.
Prof Bruce Levine, the director of the Clinical Cell and Vaccine Production Facility at the University of Pennsylvania, told the BBC: “This is a first – gene editing has not to date been used in a human trial [for HIV]. We’ve been able to use this technology in HIV and show it is safe and feasible, so it is an evolution in the treatment of HIV from daily antiretroviral therapy.”