Scientists have cured living animals of HIV using the CRISPR genome editing system. In a new study published this week, US scientists have shown they can completely remove HIV DNA from human cells implanted in mice.
Scientists have completely eradicated the virus in animals for the first time, and they are already on their way to a clinical trial in humans. The most interesting thing is that a study conducted at the School of Medicine. Lewis Katz at Temple University and the University of Pittsburgh, included a "humanized" model in which human immune cells were transplanted into mice and infected with the virus. The new work, led by Dr. Wenhui Hu, builds on previous research by the same team in which they managed to remove HIV-1 from the genome of most tissues. Now they have managed to remove the virus from all tissues.
“Our new study is more comprehensive,” said Dr. Hu. “We have validated our previous work and improved the efficiency of gene editing. We also proved that our strategy is effective in two additional models, one of which is an acute infection in mouse cells and the other is a chronic or latent infection in human cells,”the scientist added. The team tested 3 groups of mice. The first group was infected with HIV-1 and the second with EcoHIV (mouse equivalent of HIV-1 human). A third group of mice were challenged with human T cells that were infected with HIV-1. In the first group, scientists were able to genetically inactivate HIV-1, reducing the expression of RNA viral genes by 95%, which confirmed earlier results. The second group had an additional problem: the virus was actively spreading and multiplying.“During an acute infection, HIV is actively spreading,” explained Dr. Khalili. "In mice infected with EcoHIV, we were able to investigate the capabilities of CRISPR / Cas9 technology to block viral replication and potentially prevent systemic infection." CRISPR / Cas9 technology uses tags that identify the location of the mutation and an enzyme that acts like tiny scissors and cuts DNA at a precise location, allowing small portions of the gene to be removed. Scientists have eliminated 96% EcoHIV in mice. For the first time, they managed to destroy HIV-1 using the CRISPR / Cas9 system.which identify the location of the mutation, and an enzyme that acts like tiny scissors and cuts DNA at a precise location, allowing small portions of the gene to be removed. Scientists have eliminated 96% EcoHIV in mice. For the first time, they managed to destroy HIV-1 using the CRISPR / Cas9 system.which identify the location of the mutation, and an enzyme that acts like tiny scissors and cuts DNA at a precise location, allowing small portions of the gene to be removed. Scientists have eliminated 96% EcoHIV in mice. For the first time, they managed to destroy HIV-1 using the CRISPR / Cas9 system.
Finally, they come to a third animal model: humanized mice inoculated with human immune cells, including T cells, which can harbor HIV. "These animals carry latent HIV in the human T cell genomes, where the virus can lurk," explained Dr. Hu. After a single processing with the CRISPR / Cas9 genome editing system, the scientists were able to completely remove viral fragments from latently infected human cells embedded in mouse tissues and organs. The new study is another step towards curing HIV infection. "Next, we will repeat this study in primates - a more appropriate animal model." “Our ultimate goal is a clinical trial of this method in humans,” said Dr. Khalili. Currently, it is only possible to suppress the development of the disease, but not to cure it. Clinical trials of the newest method in humans may begin in a few years.