Scientists have excised HIV DNA from the genomes of three different animal models, including transgenic mice who were transplanted with human immune cells. The research, conducted by scientists at the Lewis Katz School of Medicine at Temple University and the University of Pittsburgh, was published in the journal Molecular Therapy.

This is the first study to successfully excise HIV DNA from three separate animal models and the first to demonstrate that HIV-1 replication can be shut down with the gene editing technology: CRISPR/Cas9.

“We confirmed the data from our previous work and have improved the efficiency of our gene editing strategy,” said lead author Wenhui Hu, MD, PhD. “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.” 

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The scientists demonstrated genetic inactivation of HIV-1 in transgenic mice, reducing RNA expression of viral genes by approximately 60 to 90%. The gene editing system was also tested on mice with EcoHIV, the mouse equivalent of human HIV-1, with results showing excision efficiency that reached 96%.

The third animal model employed mice that were engrafted with human immune cells and then infected with HIV-1. Latent HIV in human T cells has the ability to escape detection however, a single treatment with CRISPR/Cas9 in the infected human cells showed that the viral fragments in the mouse’s tissues and organs was successfully excised.

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