Scientists have successfully removed HIV-1 DNA from mice genome, marking a huge milestone in the search for a cure to the HIV infection in humans.
To achieve this feat, researchers from the Lewis Katz School of Medicine at Temple University and the University of Nebraska Medical Center used the gene editing tool CRISPR-Cas9 in combination with an experimental drug to clear out the virus from the animal genomes. It's an innovative technique that may be the path to finding a cure for the deadly infection, although further research will likely be necessary moving forward.
"Our study shows that treatment to suppress HIV replication and gene editing therapy, when given sequentially, can eliminate HIV from cells and organs of infected animals," said study author Kamel Khalili, PhD in a press release.
CRISPR, ART Work Together To Flush Out HIV
Current treatment for HIV makes use of a technique known as antiretroviral therapy or ART, which prevents the virus from replicating. However, it does not actually eliminate HIV from the body. HIV integrates itself into the genomes of cells in the immune system, where it can hide dormant and out of reach of antiretroviral medication, which means that it's capable of rebounding and replicating to cause the development of AIDS.
In the study published in the journal Nature Communications, the research team used a new gene editing system using CRISPR-Cas9 technology in conjunction with a recently developed technique of ART called long-acting slow-effective release or LASER ART, which targets viral pockets where the virus may be lying dormant and lowers HIV replication for extended periods of time.
Previous research by Khalili and his team showed that gene editing cannot purge HIV from genomes on its own, but by combining it with the new antiretroviral drug, the researchers hoped it would become more effective.
According to Khalili, the team wanted to see if LASER ART could suppress the replication of HIV long enough to allow the CRISPR-Cas9 to get rid of all the cells of viral DNA.
How They Did It
To see whether the combination of CRISPR and LASER ART technology would be effective against HIV, the researchers engineered mice to produce human T cells that are susceptible to HIV infection. This would allow long-term infection as well as latency induced by ART. When the mice were infected, they were treated with LASER ART followed by CRISPR-Cas9.
Findings showed that there was complete elimination of HIV DNA in roughly one-third of the infected mice.
"The big message of this work is that it takes both CRISPR-Cas9 and virus suppression through a method such as LASER ART, administered together, to produce a cure for HIV infection," Khalili said, adding that there is now a clear path for researchers to follow up with trials in non-human primates or even clinical trials on humans within the year.