Researchers say they have removed HIV from the DNA of mice, an achievement the scientists say could be an early step toward an elusive cure for humans.
The breakthrough, detailed earlier this week in a studycredited to more than 30 scientists from Temple University and the University of Nebraska Medical Center, was made possible by an antiviral drug in combination with the tool called CRISPR that can edit genes. The researchers eliminated HIV in nine of 23 mice that were modified so their immune systems better mimicked those of humans.
Clinical trials for the gene-editing component of the cure could start as early as next year if the Food and Drug Administration approves them, said Kamel Khalili, one of the study’s senior investigators. But he and other HIV experts emphasized that there is a big scientific leap from promising results in mice to success in humans.
Earlier this year, revelations that a second person had seemingly been rid of the virus raised hopes that another patient’s cure 12 years earlier was not a one-off victory. But scientists cautioned that it was too early to declare the anonymous second patient cured — and that, regardless, the case did not herald a widespread cure for the devastating condition. Both patients were treated with stem cell transplants, which experts say are risky, bring serious side-effects and would not be preferred for most patients.
Previously, Khalili’s team at Temple had found a way to remove significant amounts of HIV DNA from rats and mice. But the technique could not completely remove the infection. So Khalili’s lab joined forces with a University of Nebraska Medical Center lab attacking the problem in a different way. Together, the scientists combined the gene-editing strategy with a drug designed to beat back HIV.
Howard Gendelman from UNMC told The Post that his team’s experimental drug is engineered to act over a longer time than normal therapies, meaning it can be administered every couple of months instead of every day. It is also better able to target HIV in the body, he said. It is crucial that gene editing remove every last bit of HIV, he said, and the drug makes that task easier.