Temple Univ. researchers use CRISPR to treat HIV in mice
Illustration by Giovanni Curcio / The Burn-In

Researchers from Temple University recently announced promising news in the continuing quest for an HIV cure, the virus responsible for AIDS, as Discover reports.

Almost 37 million people worldwide have HIV or AIDS. For years doctors have utilized antiretroviral therapy, or ART, to treat the virus. But while ART has extended the lives of many people with AIDS, HIV remains in the body.

These facts make the Temple report exciting. The team, led by virologist Kamel Khalili, used a new form of ART, dubbed LASER ART, in conjunction with CRISPR-Cas9 gene editing to successfully remove the HIV from a group of mice. Khalili spoke optimistically about the breakthrough’s future implications for HIV and AIDS treatment in humans:

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“We now have a clear path to move ahead to trials in non-human primates and possibly clinical trials in human patients within the year.”

LASER to the Rescue

The journey began with researchers discovering how to simulate HIV infection in mice. To do this, they created “humanized mice,” mice that make human immune cells rather than mice immune cells. The mice then became susceptible to HIV. After they received the infection, scientists saw the virus attack the usual places: lymph nodes, lungs, liver, spleen, and brain.

The researchers then applied the LASER ART treatment. LASER stands for “long-acting, slow effective release.” While standard ART therapy necessitates daily doses, LASER ART only requires a weekly administration using “retroviral nanoparticles” to stop HIV from replicating. Notably, the LASER ART treatment did its job, preventing 99 percent of the virus from reproducing. However, LASER ART still could not remove the virus altogether. This is where CRISPR came in.

CRISPR Comes Through

After the LASER ART therapy, the Temple team used CRISPR-Cas9 to edit out the HIV DNA from the genomes of the infected mice. The combination knocked out any vestige of the virus in 40 percent of the mice. The team reported their findings in the Nature Communications journal. “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,” Khalili said.

Related: Is CRISPR the key to finding a cure for HIV?

While the results are promising, big questions still exist. For instance, why did the LASER ART and CRISPR combination only work in 40 percent of the mice? Jonathan Stoye of the Francis Crick Institute in London, who was not part of the study, posed this question. But Stoye was still optimistic about the breakthrough. “This is a tremendously exciting paper offering a vision of a potential path to a permanent cure to AIDS in the future,” Stoye said.

Indeed, the results from the Temple researchers are promising. So much time and effort have gone into eradicating HIV and AIDS. The Temple team’s endeavors certainly seem to have brought the world one step closer to finding a cure for AIDS.

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