This is huge.
Researchers at Temple University in Philadelphia say they have found a safe way to eliminate the HIV-1 virus from CD4+ T cells through a gene editing system.
“The findings are important on multiple levels,” Kamel Khalili, professor and chair of the Department of Neuroscience at Temple, says in a statement. “They demonstrate the effectiveness of our gene editing system in eliminating HIV from the DNA of CD4+ T-cells and, by introducing mutations into the viral genome, permanently inactivate viral replication.”
“Further,” Khalili continued, “they show that the system can protect cells from reinfections and that the technology is safe for the cells, with no toxic effects.”
The findings were published in Scientific Reports earlier this week and mark the first time researchers at Temple have actually been able to remove HIV from an infected T cell, opening the door for even more in-depth research.
“The questions they address are critical, and the results allow us to move ahead with this technology,” Khalili said.
In a press release, Dr. Thomas Malcolm, CEO and President of Excision Biotherapeutics, which holds the exclusive rights to commercialize the gene editing technology, said: “This is a major advance in safety and efficacy for the use of CRIPSR/Cas9 gRNA HIV eradication for us in humans. These exciting results also reflect our ability to select viral gene targets for safe eradication of any viral genome in our current pipeline of gene editing therapeutics.”
Gene editing has long been thought of as a potential cure for HIV, but scientists have feared that if not done properly, the side effects could be disastrous, even fatal. Researchers at Temple believe they have overcome this hurdle, but they are careful to note that challenges remain.
“First, it will be important to maximize elimination of viral sequences from patients,” the authors state. “This will require analysis of the HIV-1 quasi-species harbored by patients’ CD4+ T-cells and design of suitable, i.e. personalized CRISPRs. Second, improved delivery of CRISPR/Cas9 will be required to target the majority of circulating T-cells.”