HIV_2 Photo

New CRISPR/Cas9 Therapy Eliminates HIV in Animals

Scientists have managed to completely eliminate the HIV virus in living mice using the revolutionary CRISPR/Cas9 gene-editing technology. They have demonstrated how it excises the viral DNA from the host animal and prevents further infection, providing hope that it could one day benefit humans.

Despite decades of research, HIV/AIDS remains one of the most elusive diseases affecting mankind for which no definite cure exists. Although tremendous advances have been made in prevention and symptomatic treatment of HIV, roughly 37 million people remain infected according to the World Health Organization (WHO). This is mostly due to the viruses’ extremely high mutation rate and its ability to remain latent for years before striking at a time of vulnerability.

Recently, a research collaboration from the Lewis Katz School of Medicine at Temple University (LKSOM) and the University of Pittsburgh have shown that the future for HIV treatment could lie in the breakthrough gene-editing technology known as CRISPR/Cas 9. They used it to successfully excise the HIV-1 viral DNA from infected transgenic mice and completely shut down its replication potential. The study is set to be printed in May’s issue of Molecular Therapy.

“During acute infection, HIV actively replicates,” study co-author Dr. Khalili explained, adding “With EcoHIV mice, we were able to investigate the ability of the CRISPR/Cas9 strategy to block viral replication and potentially prevent systemic infection.”

The group’s research was based on a previous proof-of-concept study published in 2016, however, now they have expanded it substantially, making them the first to successfully treat three different mice models, including a “humanized” model with transplanted infected human immune cells. They have recorded an impressive 60 to 95 percent reduction of viral RNA in all animals tested.

“Our new study is more comprehensive,” said study lead Dr. Hu.. “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.”

Although these results are still to some extent preliminary and limited to mice, they solidified CRISPR´s role in possibly solving the HIV puzzle in the future. However, extensive research will have to be carried out on the topic before human trials can begin.

Learn more about CRISPR/Cas9 in the video below:

By Luka Zupančič, MSc, University of Applied Sciences Technikum Vienna.

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