Using gene editing, researchers may be able to alter people's cells to make them resistant to HIV, the New York Times reports. A small study in the New England Journal of Medicine from the University of Pennsylvania's Carl June and colleagues reports using zinc finger nucleases to target CCR5, which encodes a coreceptor for HIV entry.
In this pilot study, June and colleagues infused 12 HIV patients with autologous CD4-enriched T cells with CCR5 genes modified by ZFNs in a hope to mimic the resistance to the virus exhibited by people with CCR5-delta32 mutations. While this study was primarily examining the safety of such a treatment, the researchers found that after treatment interruption in half the patients and though HIV levels went up and immune cell levels went down in those patients, the modified immune cells did not decline as much, indicating that they may be protective. The half-life of the modified cells is an average 48 weeks.
One patient experienced side effects of fever, chills, and joint pain, which the researchers attributed to the transfusion rather than the treatment. Indeed that patient tells the Times that he'd go through the treatment again if it could lead him to be HIV-negative.
"It's a great strategy," Anthony Fauci, the director of the National Institute of Allergy and Infectious Diseases and who was not involved in the study, tells the Times. "It's exciting, interesting, elegant science. But a lot of 'ifs' need to be addressed before you can say 'Wow, this could really work.' "