CRISPRResearchers have shown that HIV DNA can be excised from the genomes of living animals to eliminate further infections. This is the first time a “humanised” model in mice has been used by transplanting human immune cells into mice and infecting the animals with the virus. The research was published in Molecular Therapy.

This is the first study to demonstrate that HIV-1 replication can be completely shut down and the virus eliminated from infected cells in animals with use of CRISPR/Cas9, paving the way to a human clinical trial.

 This new study builds on the same team’s previous research, where they demonstrated that the targeted fragments of HIV-1 from the genome in most tissues in the experimental animals could be deleted.

Now, a year later – they have been able to eliminate the virus from every tissue.

“We confirmed the data from our previous work and 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,” said Wenhui Hu, associate professor at the Center for Metabolic Disease Research and co-senior author of the study.

“During acute infection, HIV actively replicates. With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection,” explained Kamel Khalili, professor and chair of the department of neuroscience at LKSOM and co-senior investigator of the study.

“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 the elimination of HIV-1 DNA in latently infected T cells and other sanctuary sites for HIV-1, including brain cells,” Dr. Khalili concluded.

“Our eventual goal is a clinical trial in human patients,” he adds.