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Researchers Use CRISPR To Cure HIV In Mice

Researchers say they’re one step closer to finding a potential cure for HIV after successfully eliminating the virus in living mice for the first time.

A new study, published on Tuesday in the journal Nature Communications, details the efforts of researchers to use the gene-editing tool CRISPR to eliminate HIV from the bodies of infected mice. While it only worked about a third of the time in the experiment, the success represents “a big step forward,” said Chen Liang, a professor of medicine at McGill University who wasn’t involved in the study.

“This observation is the first step toward showing for the first time, to my knowledge, that HIV is a curable disease,” says one of the study’s lead authors, Kamel Khalili, director of the center for neurovirology and the Comprehensive NeuroAIDS Center at Temple University’s Lewis Katz School of Medicine.

Khalili, it’s worth pointing out, is the founder and principal scientific advisor of Excision BioTherapeutics, a Philadelphia-based company that uses CRISPR to treat viral diseases. Excision BioTherapeutics holds the exclusive license for the commercial application of this new therapy. Nearly 37 million people worldwide are infected with HIV-1, and more than 5,000 people are infected each day, according to UNAIDS.

So it makes sense that this team is eager to begin clinical trials, but the new research needs to be met with a hefty dose of caution. In addition to explaining the low success rate, the researchers will need to show that the CRISPR edits aren’t resulting in long-term side effects, such as cancer.

The team is already testing the therapy in non-human primates, and hoping to confirm the same results. If it can, it would open the door for human testing.