One in every hundred Europeans is lucky enough to have the CCR5-Delta 32 mutation, an alteration of some blood cells that gives them natural resistance to the human immunodeficiency virus (HIV), a microorganism that attacks and weakens the immune system and gives way to the Acquired Immune Deficiency Syndrome or AIDS , characterized by low levels of defenses and the appearance of opportunistic infections .
The mutation of these individuals prevents HIV from penetrating its “target cells”, the CD4 T lymphocytes, which are key in the body’s immune response. Understanding this phenomenon and developing a gene therapy that reproduces this change in the cells of HIV-infected people and thus cures them from the infection is the objective of a three-year project funded with 942,000 euros by a consortium made up of four Spanish entities : the IrsiCaixa AIDS Research Institute, the Higher Council for Scientific Research (CSIC) and the companies Karuna Good Cell Technologies and Praxis Pharmaceutical.
In the first phase of the project, led by the CSIC, genomic editing techniques developed by the consortium will be used to modify the CD4 T lymphocytes of HIV-infected people in vitro and make them resistant to it. These modified cells are then returned to the patient’s body, which will thus have a percentage of CD4 + T lymphocytes immune to HIV.
The researchers say that the percentage of resistant cells could grow if we start from hematological stem cells. For this reason, the second phase of the project will isolate stem cells from the patient himself, deactivate the gene and transfer them back to the patient. In this way, a permanent HIV-resistant immune system could be achieved.
During the three years that the investigations will last, both in vitro and animal studies will be carried out, which will help determine the safety and efficacy of a procedure that adds to the long list of advances that fight HIV with increasing efficacy, as demonstrated by the the fact that AIDS has gone from being a fatal disease to a controllable chronic infection.
Image (CSIC): HIV in the bloodstream.
