Research highlight

Gene therapy for AIDS

Nature Medicine

February 16, 2009

The results from the first clinical trial of gene therapy in patients infected with HIV are reported online in Nature Medicine this week.

Gene therapy is an appealing option to treat AIDS, as it has the potential to be a once-only treatment that reduces viral load, preserves the immune system and avoids lifetime antiretroviral therapy. Ronald Mitsuyasu and colleagues have now completed the first randomized, double-blind, placebo-controlled, gene transfer clinical trial in 74 HIV-infected adults.

The patients received either placebo or blood stem cells carrying a molecule called OZ1, which prevented viral replication by targeting two key HIV proteins. OZ1 was safe, causing no adverse effects over the course of the trial. Although there was no statistical difference in viral load between the OZ1 and placebo group at weeks 47 and 48 of the trial, counts of CD4+ lymphocytes?the cell population that is depleted by HIV?were higher in the OZ1 group at 100 weeks.

This study indicates that gene therapy is safe and active in people with HIV and can be developed as a conventional therapy against AIDS.

Author contact:

Ronald Mitsuyasu (University of California, Los Angeles, CA, USA)

Tel: +1 310 557 1891; E-mail:

doi: 10.1038/nm.1932

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