It deserves to be called a major breakthrough. Scientists at Temple University in Pennsylvania and the University of Nebraska Medical Center have reported curing nine mice of HIV, the first such instance in history.
They combined CRISPR gene-editing technology and an antiretroviral therapy (ART) called LASER ART to eliminate HIV in the subject mice. This combination therapy called “CRISPR-LASER ART” is now being tested in primates.
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Because of this step forward, researchers say they’re all the more closer to finding a potential cure for HIV. On average, people afflicted with AIDS live about three years after their diagnosis.
“We think this study is a major breakthrough because it for the first time demonstrates after 40 years of the AIDS epidemic that the HIV disease is a curable disease,” said study co-author Dr. Kamel Khalili.
Dr. Khalili is chair of the department of neuroscience and director of the Center for Neurovirology and the Comprehensive NeuroAIDS Center at Temple University.
HIV is currently treated with ART, which suppresses it from replicating and prevents many patients in the U.S. from developing AIDS. ART, however, doesn’t rid the body of HIV. If a patient stops ART, the virus will continue to replicate.
CRISPR-LASER ART was able to destroy the virus in “humanized” mice. These mice were injected with human bone marrow to imitate the human immune system.
The study authors used two different tools to combat the virus: CRISPR technology and LASER ART.
CRISPR-Cas9 is a marvelous gene editing tool that can help researchers treat or potentially cure genetic diseases. It gives scientists the ability to alter an organism’s DNA. CRISPR-Cas9 can add, remove or change certain genetic material.
On the other hand, LASER ART is a “super” form of ART that keeps replication of HIV at low levels for a longer period of time. The antiretroviral drug is then stored in nanocrystals, which slowly release the drug where the virus is located.
“We’re going at the root cause,” said study co-author Dr. Howard Gendelman. “We’re going after the virus that’s already integrated in the genome of the host cell.”
Dr. Gendelman is chair of UNMC’s pharmacology and experimental neuroscience department and director of the the Center for Neurodegenerative Diseases.
Researchers used the two treatments at the same time. First, they administered the LASER ART to reduce HIV growth. They then used CRISPR as “chemical scissors” to “eliminate the residual integrated HIV DNA still present.
The virus did not return in nine of the 21 mice in which the method was tested, according to Dr. Khalili.
The study authors say the findings are promising. They’re now testing the CRISPR-LASER ART combination on primates.
“Things that work in mice, may not work in men,” said Dr. Gendelman. “The limitations of any mouse work have to do with the species, how the drug is administered, the distribution, which is a lot easier than a man or a woman.”
Researchers aim to be granted approval by the U.S. Food and Drug Administration (FDA) to conduct a phase 1 clinical trial in humans by mid-2020.
“We are at the cusp of a scientific revolution, a breakthrough in human genomes that can change the course, quality and longevity of life,” said Dr. Gendelman.