Mice Cured of HIV Using CRISPR for the First Time but More Research Needed

Approximately 37 million people on Earth have HIV, with 5,000 people contracting the virus each day. With these numbers in mind, a team of scientists has been working on finding a cure for the virus, making recent significant advances. 

For the first time ever, researchers from Temple University and the University of Nebraska Medical Center (UNMC) have cured nine mice of HIV. Their findings were published on Tuesday in Nature Communications. 

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A few terms and explanations to begin with

HIV, or human immunodeficiency virus, is a virus that attacks the body's immune system, making a person more prone to falling ill. HIV can develop into late-stage HIV, or AIDS, with a life span of about three years once developed to this stage.

So far HIV can be treated with antiretroviral therapy (ART), which suppresses the virus from replicating, but is currently uncurable. Thus, the urgent need for finding a cure.

In an exciting turn of events, the team of scientists was able to cure mice of HIV by using a combination of CRISPR-Cas9 gene-editing technology, and LASER ART, a therapeutic treatment that slows down the replication of the virus for longer periods of time.

CRISPR-Cas9 is a tool used to edit genes and has been widely praised by the medical and scientific world for its treatment and potential cure for viruses. 

LASER ART is a stronger form of ART, that slows down the replication of the viruses. This is of particular use in this instance as the antiretroviral drug can be stored in nanocrystals, slowly releasing the drug where the virus is found. 

Dr. Howard Gendelman, the co-author of the study and chair of UNMC's pharmacology and experimental neuroscience department and director of the Center for Neurodegenerative Diseases, said: "We're going to the root cause, we're going after the virus that's already integrated in the genome of the host cell."

How did the team cure mice of HIV?

The mice in the test were injected with human bone marrow in an attempt to replicate the human immune system. 

By combining the treatment with CRISPR-Cas9 and LASER ART, the team had some remarkable end results. 

To begin with, the scientists administered the LASER ART to slow down the HIV growth, then they added the CRISPR treatment as "chemical scissors" to "eliminate the residual integrated HIV DNA still present," said Gendelman. 

The result?

Nine out of 21 mice were cured of HIV. 

That's not a huge number, and there are clear steps still to be taken before this can become a human clinical trial, but it's certainly a positive start. 

The team will now be testing primates, before potentially moving on to humans. Testing primates will help the research as humans and primates share closer similarities to that of humans and mice. 

Hopeful, the researchers are looking to be granted approval for a human clinical trial by the Food and Drug Administration. If all works out, could start as early as mid-2020. 

As Gendelman positively stated, "We are at the cusp of a scientific revolution in human genomes that can change the course, quality of life, and longevity of life."