Modified AIDS Virus Cures Mice of Sickle-Cell Anemia

HIV, "stripped of potentially harmful components," can deliver a "therapeutic gene" to the bone marrow to cure sickle-cell anemia in mice, reports a study in today's issue of the journal Science, the Wall Street Journal reports. The AIDS virus works well for gene therapy because of its ability "to carry a large therapeutic gene and effectively insert it into the cells' normal genetic material," senior study author and Harvard Medical School assistant professor Philippe Leboulch said (Johannes, Wall Street Journal, 12/14). Researchers are currently testing the procedure on monkeys to make sure the modified virus does not have "other harmful genetic errors" before it is tested on humans (Mishra, Boston Globe, 12/14). The modified HIV is "the single best thing we've seen in gene therapy for years," Leboulch said, noting that other viruses are not as effective at "worming their way into" cell nuclei. But the Journal notes that using HIV for therapy "will require careful safety scrutiny" and that "it will take a lot of work to convince government regulators" that the virus can be successfully used in this way (Wall Street Journal, 12/14).