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Hannah Knowles, the Washington Post
updated on July 5, 2019
Researchers say that they have removed HIV from mouse DNA, a feat that scientists say could be a first step towards a cure for humans.
This breakthrough, detailed earlier this week in a study involving more than 30 scientists from Temple University and the University of Nebraska Medical Center, was made possible by an antiviral drug associated with a tool called CRISPR allowing gene editing. The researchers eliminated HIV in 9 out of 23 mice modified so that their immune system mimics that of the human.
The clinical trials for the gene editing component of the cure could begin as early as next year if the Food and Drug Administration approves them, said Kamel Khalili, one of the study's lead researchers. But he and other HIV experts pointed out that there was a big scientific leap between promising results in mice and success in humans.
"We knew what we had to do, but the technology was not available," Khalili told the Washington Post, saying he and his team were waiting for a tool such as CRISPR to fight a virus that "goes into the tissue of our chromosomes ". With the release of genes, finally, a reality, he said, "the result has been incredible."
Once fatal, HIV can now be managed with a treatment called antiretroviral therapy. The therapy only controls the virus; without constant medication, the virus will rapidly decimate the patient's ability to fight the disease. HIV infects 37 million people worldwide, according to the latest data from the World Health Organization, and only about 22 million of these people are receiving antiretroviral therapy. According to the WHO, nearly one million people have died of HIV – related problems in 2017.
Earlier this year, revelations that a second person had apparently been cleared of the virus had raised hopes that another patient's recovery 12 years earlier was not a single victory. But scientists warned that it was too early to declare the second anonymous patient cured – and that, despite everything, the case did not announce a widespread cure for this devastating disease. Both patients have been treated with stem cell transplants, which experts say is risky, leads to serious side effects and would not be preferable 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 eliminate the infection. Khalili's lab has partnered with a lab at the University of Nebraska Medical Center to tackle the problem differently. Together, scientists have linked the gene editing strategy to a drug designed to fight HIV.
Howard Gendelman of the UNMC told The Post that his team's experimental drug was designed to work longer than normal treatments, meaning it could be administered every two months instead of every day. He is also better able to target HIV in the body, he said. It is crucial that gene editing removes HIV so far, he said, and the drug makes it easier.
"If you can reduce the amount of virus that remains for CRISPR, the likelihood that CRISPR will be effective will increase dramatically," he said.
But if Khalili's team is able to go ahead with human trials, they will use standard drugs rather than those developed by the UNMC laboratory, as they probably would not have been approved, said Khalili.
Steven Deeks, a professor at the University of California at San Francisco who has done a lot of work on HIV, said that using gene editing to eliminate HIV from a live animal is a notable progress.
But he warned that the use of the technique on humans would be much more difficult: scientists will face more variations of the virus, more difficulties to use genetic modification technology and the possibility of cutting human genes while trying to target HIV, he said. These are daunting problems, said Deeks, particularly successfully, because the virus had to be completely eliminated.
"For this approach to work, they must really destroy 100% of the genomes – you can not leave anything behind," said Deeks. "Any of them can revive the whole process."
The scientists behind the new study recognize these challenges. Khalili said the researchers are currently publishing another study on the use of gene editing to target HIV in primates and hope to provide the solution to humans. But scientists need to make sure their techniques are safe, he said. They also want to increase the chances of their methods working from the success rate of about a third that they have seen with mice.
Gendelman said that although modern drugs can fight HIV, permanent treatment would bring great benefits to patients. Constant medical treatment can contribute to other health complications, he said, and the mere fact of keeping the tablets in your medicine cabinet may prompt careful consideration by the family or family. 39, other important people.
"There is a huge stigma," he said. "Every time you take these pills, you are reminded that you have HIV."
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