HIV breakthrough: Scientists remove virus in animals using gene editing

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Scientists have managed to successfully ‘edit out’ the virus in HIV-positive mice. Worldwide, tens of millions of people are living with HIV. While scientists and medical professionals do not yet have a permanent cure for the virus, researchers have just made a breakthrough: they managed to eliminate the HIV-1 infection in mice.
According to the Centers for Disease Control and Prevention (CDC), more than 36 million people across the world are HIV positive, and approximately 1.2 million people in the United States live with the virus.
While there is currently no cure for the infection, scientists have just moved closer to finding one. Using a gene editing technology called “CRISPR/Cas9,” the researchers successfully excised the HIV-1 provirus in three animal models.
A provirus is an inactive form of virus. It occurs when the virus has integrated into the genes of a cell.
In the case of HIV, these host cells are the so-called CD4 cells – once the virus has been incorporated into the DNA of the CD4 cells, it replicates itself with each generation of CD4 cells.
The three mouse models used in the current research included a “humanized” model, in which the mice were genetically modified to have human immune cells, which were then infected with HIV-1.
The team was co-led by Dr. Wenhui Hu, Ph.D., associate professor in the Center for Metabolic Disease Research and the Department of Pathology at the Lewis Katz School of Medicine (LKSOM) at Temple University in Philadelphia, together with Kamel Khalili, Ph.D., Laura H. Carnell Professor and chair of the Department of Neuroscience at LKSOM, and Won-Bin Young, Ph.D, who just recently joined LKSOM.
The new study – published in the journal Molecular Therapy – builds on previous research by the same team, during which they used genetically modified rodents to demonstrate that their gene editing technology could eliminate the HIV-1-infected segments of DNA.
“Our new study is more comprehensive,” Dr. Hu explains. “We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy.
We also show that the strategy is effective in two additional mouse models, one representing acute infection in mouse cells and the other representing chronic, or latent, infection in human cells.”
Dr. Hu and team inactivated HIV-1, significantly reducing the RNA expression of viral genes in the organs and tissues of genetically modified mice. Specifically, the RNA expression was reduced by approximately 60 to 95 percent.
The researchers then tested their findings by acutely infecting mice with EcoHIV – the equivalent of the HIV-1 in humans. Dr. Khalili explains the procedure:
“During acute infection, HIV actively replicates. With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection.”

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