Important note: Information in this article was accurate in 1995. The state of the art may have changed since the publication date.
Newsday - February 4, 1995
Laurie Garrett. Staff Correspondent
Early results of the first genetic engineering experiment on HIV-positive human beings were presented last week by the University of Michigan's Dr. Gary Nabel, at the second National Conference on Human Retroviruses in Washington, D.C.
At least some mutant HIV genes reached their targets, Nabel reported, and are producing a phony version of a protein essential to HIV replication. He said he hopes to have concrete results by July. So far, the patients have experienced no side effects, Nabel said. None of the three participants had developed full-blown AIDS when the study began.
Nabel's team worked with a mutant form of an HIV gene called "rev." This gene makes a protein needed for the packaging and release of new viruses. But the mutant gene won't do that job. It is intended to act as a decoy, taking the place of the normal gene and thus, mucking up the viral machinery.
To introduce the mutant genes into the patients' systems, the University of Michigan researchers coated microscopic beads of gold with sticky strands of the mutant DNA. After drawing plasma from the patients, they bombarded the white blood cells with the tiny "bullets," as Nabel called them, using a high pressure device. White blood cells, or lymphocytes, are the main target of HIV infection.
About a billion bombarded, genetically engineered cells were then put back into each patient. Months later, two patients got a second round.
The work is very preliminary, only months into a process that will eventually involve 12 patients. Six will have their cells bombarded with gold beads. The other six will have the same mutant genes carried into their cells aboard modified harmless viruses. In essence, they will be "infected" with the mutant gene. Both groups of patients will also be treated with drugs intended to boost their immune systems.
Nabel's approach to gene therapy for HIV is one of many currently under development around the world.
Because the AIDS virus exerts its lethal effects by inserting itself into human genes, Nabel said, "HIV is a genetic disease. And it ought to be possible to tackle it by genetic means."
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