A dual therapy, based on the use of Crispr technology, has just been proven in mice. With these advances, it is a promise of therapy, which would be able to eradicate the disease everywhere in the world, which is taking shape.
Hervé Poirier, editor-in-chief at the magazine Epsiloon, we talks today about a gene therapy that seems poised to eradicate AIDS. Four cases of HIV remission (human immunodeficiency virus), observed in recent years, based on much more cumbersome methods, demonstrate that the strategy does indeed work in humans.
franceinfo: Another piece of information on AIDS research that needs to be explained and on which we need to remain cautious?
Herve Poirier: Yes, of course, but one can imagine that a few intravenous injections could soon be enough to eradicate forever all traces of the virus in AIDS patients. This therapy exploits a natural mutation, present in less than 1% of northern Europeans. The mutation codes for a protein in the cell membrane which constitutes the main access door of the virus. With the mutated form, HIV runs into a wall. The rare people who are lucky enough to inherit it are therefore naturally immune.
The principle of the therapy consists of injecting a harmless virus into the blood of the sick, which will penetrate into the nucleus of the cells – we are mainly targeting those of the immune system. Thanks to the famous genetic scissors, CRISPR-Cas9, this virus is programmed to register the life-saving mutation directly in the patient’s genome.
And it works ?
A first clinical trial started in the United States last fall, on a single patient. And an American team has just presented a test that goes even further: the injected virus not only causes the right mutation, but also eliminates all traces of AIDS in the genome – remember that AIDS is inserted in the DNA infected people, which can cause reinfection.
This double gene therapy seems effective: just a few weeks after the injection, the virus was undetectable in the blood of 58% of the rodents. And the researchers are all the more confident, as the contribution of the mutation has already been proven in 5 people, who have benefited from a bone marrow transplant. Except that this last technique, very cumbersome, cannot be generalized to all patients. Gene therapy, yes.
Modifying your genome to treat yourself, is it still a little scary?
It sounds a bit like science fiction, but that’s the principle of gene therapy. And you’re right: there are still questions about the long-term safety of these genetic modifications. But this therapy could be a game-changer.
We are in fact at a pivotal moment in the fight against AIDS. 40 years after the start of the epidemic, research has made giant strides. Triple therapies are increasingly effective. Very powerful antibodies are coming. Except that of the 40 million people affected, 10 million do not have access to these treatments, particularly in Africa, due to a lack of means and structures. Inexpensive, easy to implement, gene therapy could really put an end to the epidemic.