Gene Therapy for HIV Infection?

The finding that a mutation in the CCR5 gene renders CD4 cells resistant to HIV, and the apparent cure of an HIV-infected person — the “Berlin patient” — who received an allogeneic stem-cell transplant from a donor who was homozygous for the CCR5-Δ32 deletion (NEJM JW AIDS Clin Care Feb 13 2009), led investigators at the University of Pennsylvania to explore the safety of targeted genome editing in humans with HIV infection. The study was partially supported by industry.

Twelve HIV-infected patients with virologic suppression on antiretroviral therapy (ART) were enrolled — six “immune responders” with CD4 counts >450 cells/mm³ at screening and nadirs ≥300 cells/mm³ and six “nonresponders” with CD4 counts between 200 and 500 cells/mm³ at scre…