Gene Therapy to attack HIV

Gene Therapy

Researchers say they have used gene therapy to interrupt the replication of the virus in cells taken from infected patients.

These results suggest that, with further work, this technique may keep HIV-infected patients free of disease symptoms,'' said study senior author Dr. Wenzhe Ho of The Children's Hospital of Philadelphia, in Pennsylvania. The findings are published in the journal Gene Therapy. Ho's team focused their efforts on HIV's 'tat' gene, essential for the replication of the virus within cells.
In gene therapy, genetic material is introduced into a patient's cells to take over the function of a faulty or missing gene.

The researchers developed an 'antitat' gene, designed to disable proper tat function. In experiments conducted in the laboratory, they then used a (harmless) mouse retrovirus to 'piggyback' antitat into HIV-infected laboratory-cultured cell lines, as well as immune cells taken from HIV-infected patients.

The result? ``The antitat gene inhibited HIV activation and replication'' in both the cultured cell lines and the patients' immune cells, according to the hospital statement. The gene therapy also bolstered the survival of CD4+ T lymphocytes, important immune cells that are also the favorite target of HIV.

While the technique worked in cells in the lab, the Philadelphia team stresses that much more work needs to be done to see if it can successfully suppress HIV activity in humans.

This Research could signal the advent of a whole new method of treating HIV. Today, many - but not all - patients infected with the virus can reduce their viral load to near-undetectable levels using powerful combination drug therapies. These therapies are very expensive, however, and carry a risk of side effects. Furthermore, HIV usually rebounds to pre-treatment levels once patients go off their medication.