For the first time, scientists have successfully cut HIV out from human cells, marking an advance that researchers on the project called a possible "game changer" for the disease that afflicts millions worldwide.
The researchers from Temple University found a way to eradicate HIV-1 genes, which inserts itself into an infected individual's own DNA, permanently from human cells in culture. Here's how they achieved it, according to the university's news release:
When deployed, a combination of a DNA-snipping enzyme called a nuclease and a targeting strand of RNA called a guide RNA (gRNA) hunt down the viral genome and excise the HIV-1 DNA. From there, the cell's gene repair machinery takes over, soldering the loose ends of the genome back together – resulting in virus-free cells.
To ensure the technique did not accidentally cut out any portions of the human genome, the researchers picked sequences that are unique to the HIV-1 genome and are not found in human DNA.
"This is one important step on the path toward a permanent cure for AIDS," Dr. Kamel Khalili, director of Temple's Comprehensive NeuroAIDS Center, said. "It's an exciting discovery, but it's not yet ready to go into the clinic. It's a proof of concept that we're moving in the right direction."
These findings could someday lead to a cure that would give the more than 33 million people living with HIV-1 worldwide the ability to drop a life-long regimen of antiretroviral drugs. In the U.S., the rate of HIV diagnoses fell by one-third each year within the last decade, but 1.1 million are still thought to be infected in the country.
Even with antiretroviral therapy, patients are still susceptible to other health implications, including the weakening of heart muscle, bone disease, kidney disease and more.
"These problems are often exacerbated by the toxic drugs that must be taken to control the virus," Dr. Khalili said.
Going forward, Dr. Khalili and his colleagues will need to figure out a way to ensure that the HIV-targeted therapy can make its way into all infected cells. The university also pointed out that with the potential for mutations in the HIV-1 genome, treatment might need to be customized to individual patients.
"We are working on a number of strategies so we can take the construct into preclinical studies," Dr. Khalili said. "We want to eradicate every single copy of HIV-1 from the patient. That will cure AIDS. I think this technology is the way we can do it."
Watch Dr. Khalili talk about this research:
These findings were published this week in the Proceedings of the National Academy of Sciences.
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