When it comes to research on HIV and AIDS treatments, it can be hard to know when to celebrate a small advance–everyone wants to see progress, but so many experimental avenues that seemed promising have turned out to be dead ends. Still, a new study that tried a sophisticated form of gene therapy as an HIV treatment seems cause for cautious optimism. If it bears out under further testing, the technique could lead to a one-shot, long-lasting treatment that could replace the punishing regimen of daily medications.
Treating HIV currently comes down to managing the viral load with a mixture antiretroviral drugs. Researcher John Rossi and his colleagues tried to craft a more direct treatment by genetically modifying the HIV-infected patients’ own blood stem cells and increasing the cells’ ability to fight off the virus. The researchers weren’t able to truly combat the virus in this experiment–the patients’ viral loads remained the same–but their work moved beyond previous attempts in two ways: They successfully modified blood stem cells by giving them anti-HIV genes, and those cells survived for two years in patients.
Earlier clinical studies the group conducted with the same strategy made little headway, but now the researchers have overcome two key obstacles, says Rossi, a molecular geneticist. One is that they managed to stitch the anti-HIV genes into a high percentage of the appropriate stem cells. The other is that the cells lived for a long time. “If we could increase the number of modified cells by 10- or 100-fold, we might be able to stop the virus itself,” says Rossi. [ScienceNow]

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