Gene therapy of HIV infection
American researchers report positive results of the second stage of clinical trials of a gene therapy method for the treatment of HIV infection. 74 volunteers with HIV infection participated in a double-blind placebo-controlled study.
Researchers from the University of California at Los Angeles used hematopoietic cells for the treatment of HIV infection, in the DNA of which a gene encoding enzymatic RNA (ribozyme) OZ1 was embedded. This molecule is able to block the reproduction of the human immunodeficiency virus when it enters the cell.
Half of the volunteers were injected with stem cells with a DNA site encoding the OZ1 rhizbozyme, the rest of the subjects were injected with a placebo. Scientists expected that ribozyme-equipped hematopoietic stem cells would give rise to new generations of blood cells that would be invulnerable to HIV infection.
During the first 48 weeks after the start of the experiment, the virus concentrations in the blood of the injected patients remained at the same level as in the control group. However, by the 100th week, the treated participants began to decrease their viral load, in addition, they had an increase in the number of CD4 lymphocytes - cells that are the main target of the immunodeficiency virus.
However, the results of genetic studies of blood samples of participants showed that over time the number of genetically modified cells in their blood decreased. In the fourth week after the experiment, cells with the OZ1 ribozyme were detected in 94 percent of those who received injections, on the 48th - only 12 percent, on the 100th – only seven percent.
According to the researchers, gene therapy of HIV infection did not cause significant adverse reactions in patients. In the future, such procedures can be used for additional HIV therapy along with traditional drug treatment, scientists believe.
The report on the experiment is published in the journal Nature Medicine.
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