BREAKING: Scientists Successfully Edit HIV Out of T Cells
By George, they’ve done it.
Scientists at Temple University have successfully snipped HIV-1 right out of cultured CD4+ T cells using the gene editing system that they designed. Even more importantly, it appears so far that they did so safely.
“The findings are important on multiple levels,” said Kamel Khalili, professor and chair of the Department of Neuroscience at Temple, in a news release. “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, they show that the system can protect cells from reinfections and that the technology is safe for the cells, with no toxic effects.”
It marked the first time the experiments had gone so far as to remove HIV from an infected T cell. “But the questions they address are critical, and the results allow us to move ahead with this technology,” Khalili said.
The findings were published Monday in Scientific Reports. The research was performed on blood taken from people with HIV who were not antiretroviral therapy.
In a news release issued by Excision Biotherapeutics, which holds the exclusive rights to commercialize the technology, the company’s CEO and President Dr. Thomas Malcolm 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.”
Go ahead and say the C word
Gene editing has been bandied about as a possible cure for HIV for quite some time now. But the fear has been that if genes are not edited properly and put back inside the body, the side effects could be disastrous and even fatal.
Last month, HIV Equal reported on another method of gene editing being tested in Germany called Brec1. Brec1 scientists also hope a small clinical trial with human patients can begin sometime in the future.
But for now, Temple’s ex vivo (outside the body) experiment was a huge step forward for CRISPR/Cas9.
“Our method of HIV-1 DNA excision had no adverse effects on proximal or distal cellular genes and showed no impact on cell viability, cell cycle progression or proliferation, and did not induce apoptosis, thus strongly supporting its safety at this translational phase, by all in vitro measures assessed in cultured cells,” the authors wrote in the paper. “The complete absence of genomic and off-target functional effects in all assays also provides critical support for the promise of developing this approach for future therapeutic applications.”
But 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.”
Hurdles notwithstanding, it’s an approach where attaching the word “cure” is becoming anything but sensational. “In summary, our novel ex vivo findings that our lentiviral delivery-based approach reduced HIV-1 DNA copy numbers and protein levels in PBMCs of HIV-1 infected patients provides strong proof-of-concept evidence that CRISPR/Cas9 can be effectively utilized as part of HIV Cure strategies.”