Temple University researchers say they've eliminated HIV from living animals

PHILADELPHIA (WPVI) -- Researchers at Temple University's School of Medicine and a team at the University of Nebraska Medical Center say they have, for the first time, eliminated the DNA of HIV-1, the virus responsible for AIDS, from the genomes of living animals.

The technique involves 2 new technologies: gene editing known as CRISPR and a therapy known as LASER ART, or long-acting slow-effective release anti-retroviral therapy.

The Temple scientists have previously used CRISPR-Cas9 to remove HIV DNA from genomes containing the virus.

In mice and rats, they showed that gene editing large fragments of HIV DNA from infected cells could significantly suppress active infection and replication of the virus.

LASER ART is a longer-lasting version of the antiretroviral drugs which have helped save millions of lives. Antiretroviral drugs decrease but don't entirely eliminate HIV in the body. They have to be taken for life, or the virus will rebound.

LASER drugs are made with nanocrystals, which easily move into the tissue where HIV hides and replicates. The nanocrystals can stay in tissue for weeks, slowly releasing their drug. It keeps that HIV reproduction at low levels for long periods of time, so standard antiretroviral drugs don't have to be given as often.

But neither CRISP-Cas9 nor antiretroviral drugs alone can completely get rid of HIV.

The Temple and Nebraska teams wanted to see if combining their techniques would work.



"We wanted to see whether LASER ART could suppress HIV replication long enough for CRISPR-Cas9 to completely rid cells of viral DNA." said Dr.Kamel Khalili, director of Temple's Comprehensive NeuroAIDS Center.

In the lab, specially-engineered mice were infected with HIV, then treated first with LASER ART, then CRISPR-Cas9.

At the end of the treatment period, HIV DNA had been eliminated in about a third of the infected mice.

"The big message of this work is that it takes both CRISPR-Cas9 and virus suppression through a method such as LASER ART, administered together, to produce a cure for HIV infection," said Dr. Khalil. "We now have a clear path to move ahead to trials in non-human primates and possibly clinical trials in human patients within the year."
The scientists say it puts a cure for HIV/AIDS one step closer, although it has to be replicated on a larger scale, and with humans.

Dr. Howard Gendelman, who led the Nebraska team, still remembers when he first learned about HIV.

"I was a young intern in New York City in 1981 and ' 82 where we actually saw the first cases of a disease that we really didn't understand," said Dr. Gendelman.

"We never thought even with the vaccines and the trials, for so many years that HIV could be eliminated, but today things have changed," he continues.

Doctors hope to replace the daily drug therapy of multiple pills with one or two injections.

"We figured out a way to deliver a single dose of the medicine and have it active for months. And potentially, for up to a year," he says.

The research was funded with several grants, including one from the National Institutes of Health.
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