Scientists today report initial results from humans on the safety and tolerability of a novel strategy to curb HIV disease by removing key cells from HIV-infected individuals, genetically modifying the cells to resist HIV infection and returning them to those individuals. The basic and pre-clinical research on this strategy, which eventually might help people control the virus without drugs, was funded by the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health. The Phase I clinical trial was funded by Sangamo BioSciences and was led by NIAID grantee Carl H. June, M.D., with co-investigators Bruce L. Levine, Ph.D., and Pablo Tebas, M.D., all of the Perelman School of Medicine at the University of Pennsylvania, Philadelphia.
Four weeks later, in a planned interruption of anti-HIV therapy, half the study participants stopped taking their antiretroviral drugs for 8 to 12 weeks. Investigators found that the experimental treatment was generally safe, and that the genetically modified cells appeared to be protected from HIV infection. In one volunteer who naturally had the desired mutation in half of his CCR5 genes, the virus became undetectable in his blood during the 12-week treatment interruption. Future research will include evaluating this experimental treatment in more volunteers, as well as maximizing the frequency of CCR5 disruption by ZFNs and increasing the persistence of the genetically modified cells in the body to achieve a therapeutic effect.
P Tebas et al. Gene editing of CCR5 in autologous CD4 T-cells of persons infected with HIV. The New England Journal of Medicine DOI: 10.1056/NEJMoa1300662 (2014).
NIAID Director Anthony S. Fauci, M.D., is available for comment.
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