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The study proved the effectiveness and safety of gene therapy for HIV infection
Last reviewed: 16.10.2021
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American scientists from the University of California, Los Angeles, have proven the safety of gene therapy for HIV infection. The study was conducted by specialists led by Ronald T. Mitsuyasu. T-lymphocytes are the main targets for the immunodeficiency virus. The virus penetrates into the cell due to the presence on its surface of a specific receptor, encoded by the CCR5 gene.
The method of gene therapy for HIV infection is based on the elimination of this gene from human T-lymphocytes, after which an injection of mutated cells is performed. During the treatment, about 1/3 of the T lymphocytes received a mutated CCR5 gene, which made the cells virtually invulnerable to the immunodeficiency virus.
Scientists conducted 2 studies, in which 15 people agreed to participate. Patients underwent medical supervision for 1 year after the introduction of mutated T-lymphocytes. Throughout the year, all patients had a stable growth of T-lymphocytes, in three - the viral load decreased. In addition, one patient was unable to confirm the presence of HIV in the blood.
The project manager explained this by the fact that this patient already had one copy of the mutant gene CCR5, so after the introduction of modified T-lymphocytes, he had twice as many cells insensitive to the virus.
The results of the study showed that gene therapy for HIV infection is effective and safe for humans. Modified T-lymphocytes persisted in the blood of participants for at least a year.