AIDS gene therapy: an encouraging result
Gene therapy of HIV infection was tested on six patients
Copper newsThe American biotech company Sangamo Biosciences announced the successful results of trials of the HIV gene therapy method started 2 years ago, AP reports (Gene therapy raises hope for a future AIDS cure).
The new technique consists in changing the genome of immune system cells that are the main targets of the immunodeficiency virus.
The basis of the concept of the new treatment method was the case of the recovery of "lucky Tim" – a German HIV-infected patient after a bone marrow transplant. The donor whose bone marrow was transplanted to the patient was a carrier of the CCR5 gene mutation, which makes it difficult for the immunodeficiency virus to penetrate into T-lymphocytes.
During an experiment conducted in the USA, American scientists decided to "edit" the genes of the immune system cells of volunteers with HIV infection. To do this, the researchers removed the CCR5 gene from T-lymphocytes isolated from the patients' blood. After that, the modified cells were multiplied in the laboratory, and then introduced into the body of the subjects.
Three of the study participants received approximately 2.5 billion modified lymphocytes, and three more received twice as many cells. Three months after the procedure, the number of modified cells increased significantly in the blood of 5 out of 6 subjects. As a result, approximately 6 percent of patients' T-cells were immune to HIV infection. Theoretically, in the future, this may lead to a slowdown in the development of AIDS in HIV carriers.
No significant side effects of treatment were detected during the follow-up period. The only noted reaction to the introduction of modified cells were flu-like symptoms, which were observed on average for two days.
As the authors of the study emphasize, their experiment is the first stage of a long-term study. In addition, different types of HIV use different pathways to enter the cell, so even theoretically removing the CCR5 gene may not help all patients.
Additional experiments will be required to confirm the safety of the proposed method of treating HIV infection.
In particular, scientists will have to find out how the CCR5 gene removal technique they used will affect the functions of modified cells (it is now known that the congenital absence of this gene does not affect human health). It is also unknown how long the modified cells will be able to persist in the body of patients. Finally, another problem is the assessment of the cost of the proposed treatment.
Portal "Eternal youth" http://vechnayamolodost.ru02.03.2011