Could HIV Deliver Gene Therapy Into Cells?
HIV is dangerous because of its devious ability to enter our cells and, once there, to insert its genetic material into our own. However, researchers at Harvard and MIT have taken advantage of that and used HIV to deliver a therapeutic gene to the bone marrow, where blood cells are made. It’s only been done in mice so far, but the treatment has cured the mice of sickle-cell anemia. Such a delivery technique could provide treatments for hundreds of blood-cell disorders in humans, including leukemia.
To use HIV like a UPS truck, the researchers first altered it so it couldn’t harm the mice, which have been genetically given human immune systems and sickle-cell trait; then they gave the HIV a gene that helps blood cells keep their normal shape. The modified HIV was introduced into the mice’s bone marrow, where it delivered its treatment gene into the mice’s DNA. The result: normal, round blood cells were produced instead of deformed sickle cells. Much more study is necessary, but continued success could put a human trial only three years away.
Anyone who thinks that AIDS research is over-funded is way off base. Much of “AIDS research” is actually basic science dealing with immunology and virology, with benefits far beyond those who suffer from HIV. If there is a silver lining to the very black cloud of the AIDS epidemic, this is it: medical developments useful to us all.
(For details, see the journal Science, vol. 294, no. 5,550.)
This article appears in March 15 • 2002.