Researchers from Temple University in Philadelphia said they used a long-acting, experimental HIV therapy and combined it with a gene-editing method to get rid of the virus from one-third of mice with the genetic code.
The study, which was published in the Nature Communications journal, was conducted with the help of researchers from the University of Nebraska Medical Center. These researchers have been working on creating a long-active, slow-release version of the present antiretroviral therapy. The drug could allow for a once-a-month HIV treatment, which would help in improving patients’ ability to stick to the drug regimen.
Though two-thirds of the mice with HIV had some latent infection, researchers claim it’s the first study to eradicate the disease in living animals. It’s a crucial step toward getting a cure for HIV. An HIV cure has been rather obscure since the virus stays hidden within the body’s organs. With the help of antiviral drugs, the replication of HIV is slowed down. However, if the drugs quit working, the virus can once again attack the body’s immune system.
Temple Department of Neuroscience Chair Kamel Khalili was the study’s senior investigator. She has spent five years working with the gene-editing technology – CRISPR-Cas9 – in an effort to stop the HIV replication process. It works by taking out huge parts of viral DNA from the cells of infected animals.
However, like the antiviral drugs, it alone is not enough in getting rid of latent HIV.
Khalili, with the new study, got together with scientist Howard Gendelman, who works at the University of Nebraska as an infectious disease and pharmacology researcher. Gendelman’s team has been developing a long-acting HIV therapy that targets and suppresses HIV replication in just one dose for long periods of time. The drug, which includes nanocrystals, releases slowly for weeks.
Khalili said they wanted to know if the long-acting drug had the ability to suppress HIV replication to the point that the CRISPR-Cas9 would eliminate the viral DNA cells entirely.
This latest news is regarded as a revolutionary breakthrough in the tools to fight HIV, but the CRISPR technology is still in its infancy, and there are some obstacles it will need to overcome. The University of Pennsylvania began the first human trial in the U.S. using the technology to trigger immune cells in fighting certain cancers. Since that time, there have been other approved human trials.
Khalili said the next HIV research step is to use the combination of the two in non-human primates (monkeys). He said he’s set up an appointment to talk with the U.S. Food and Drug Administration about setting up human clinical studies using the approach.