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Researchers eliminate HIV in mice through gene editing

Temple University and University of Pittsburgh scientists have taken a big step toward a cure for HIV infections using CRISPR gene-editing technology.

Eric Mack

Scientists say they've used a gene-editing technique to cure mice of HIV.

Researchers at Temple University in Philadelphia and the University of Pittsburgh said they used the CRISPR gene editing technology to remove HIV DNA from the genetic code of mice, eliminating the disease for the animals. The findings were published this week in the journal Molecular Therapy. The study builds on years of research that initially showed that the AIDS virus and HIV could be cut from cells in lab dishes.

The latest findings offer hope for a cure to HIV, which has proven an elusive virus to fight. Antiviral drugs have been used to suppress replication of the virus, but once the drugs are stopped, the virus can break out again.

The next stage for researchers is to repeat and replicate the study in primates.

"Our eventual goal is a clinical trial in human patients," said Temple researcher Kamel Khalili in a press release.

This is the first study to use the CRISPR gene-editing technology to halt HIV replication and shut down the virus in animal cells. CRISPR technology is still relatively new and has never been used to edit human genes. Researchers at University of Pennsylvania in Philadelphia have gotten preliminary approval from a federal advisory panel to run a CRISPR trial to alter immune cells to fight cancer. The US Food and Drug Administration must still approve the trial.

Many in the scientific community view CRISPR as a potentially revolutionary medical tool. But even the creators of the technology caution moving too quickly toward human trials. In 2015, CRISPR co-creator Jennifer Doudna explained in a TED talk why she and some of her colleagues have called for a "global pause" in using CRISPR for clinical applications until some of the ethical questions surrounding gene editing in humans can be explored.

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