For the first time, a team of scientists from Temple University along with researchers from the University of Nebraska, led by Kamel Khalili, have managed to eliminate the HIV virus, the virus that causes AIDS, in the genomes of live animals, an achievement fundamental to be able to develop a cure for this disease that every day affects more people in the world.
The study, published in Nature Communications, is based on developing genetically modified mice to produce human T cells susceptible to HIV infection. Once infected, a therapeutic strategy known as long-acting slow-release antiretroviral therapy, or also called LASER ART, was used to suppress the replication of the HIV virus in these animals.
The last step was to use the CRISPR gene editing technique to remove HIV DNA from the infected cells. The results showed that approximately one third of the animals stopped showing signs of the virus. The next step is to test the technique in non-human primates, and if positive results are obtained, human trials would begin, says Kamel Khalili.
However, although the team is optimistic, it is also aware that there are many differences and much ground to cover between mice and humans.
“What works in mice may not work in humans – explains Khalili in a statement -. The limitations of any work with mice have to do with the species, how the drug is administered, the distribution of these, the time, the side effects … The great message of this work is that both CRISPR is needed as the suppression of the virus through a method like LASER ART, administered all together, to produce a cure for HIV infection. Now we have a clear path to move towards trials in non-human primates and possibly clinical trials in human patients the same year. “