There is hope for the more than 36 million people across the world are HIV positive. While there is currently no cure for the infection, scientists have just moved closer to finding one. Using a gene editing technology called “CRISPR/Cas9,” the researchers successfully removed the HIV-1 provirus in three animal models.
A study team at Temple University led by Wenhui Hu demonstrated the ability to shut down HIV-1 replication in three different live animal models. The university researchers removed HIV-1 from human T-cells.
As a retrovirus, HIV has proven resistant to cures because it introduces replicating copies of itself into the genomes of its host cells. Current antiretroviral drugs can contain the virus and prevent the onset of AIDS, but how to permanently eradicate the infection is still an elusive goal for medical researchers.
“Our new study is more comprehensive,” Dr. Hu said.
“We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy. We also show that the strategy is effective in two additional mouse models, one representing acute infection in mouse cells and the other representing chronic, or latent, infection in human cells.” The research, published in the journal Molecular Therapy.
In the first model, the RNA expression of viral genes was reduced 60 to 95 percent as shown in the previous study. In the EcoHIV mice, excision efficiency reached 96 percent, marking the first evidence that this method can eradicate the virus. The third model took just one CRISPR/Cas9 treatment to excise viral fragments of latently infected human cells that were embedded in the mice.
“The next stage would be to repeat the study in primates, a more suitable animal model where HIV infection induces disease, in order to further demonstrate elimination of HIV-1 DNA in latently infected T cells and other sanctuary sites for HIV-1, including brain cells. Our eventual goal is a clinical trial in human patients.” Said Kamel Khalili, Ph.D, a lead researcher