Gene Therapy May One Day Prevent AIDS–Related Brain–Cell Death

Scientists have shown that gene therapy has potential for treating brain pathology triggered by the human immunodeficiency virus (HIV), which causes AIDS. Although brain damage is among the most common complications of AIDS, there is no recognized therapy for preventing the underlying HIV-induced cell death.

NIMH-funded researchers Dr. D.S. Strayer and colleagues from Thomas Jefferson University loaded a harmless type of virus with genes engineered to produce detoxifying enzymes, and allowed the virus to carry the loaded genes into brain cells exposed to a toxic substance released by HIV. When the genes took hold in both human brain cells in lab dishes and in brain cells in living rats, the genes began producing the beneficial enzymes, and the rate of brain-cell death dropped dramatically. Results were reported online ahead of print in Gene Therapy, on July 27, 2006.

The toxic substance produced by HIV is called gp120; it triggers molecular machinery in brain cells that causes them to self-destruct. The enzymes the scientists introduced are known to neutralize the enablers of this cell-death machinery, such as free radicals.

Agrawal L, Louboutin J-P, Reyes BAS, Van Bockstaele EJ, Strayer DS. Antioxidant Enzyme Gene Delivery to Protect from HIV-1 Gp-120-Induced Neuronal Apoptosis. Gene Therapy, E-pub ahead of print; July 27, 2006.