“In a proof-of-concept study, we show that our gene editing technology can be effectively delivered to many organs of two small animal models and excise large fragments of viral DNA from the host cell genome,” said Professor Kamel Khalili, who led the study. This, scientists hope, could one day lead to the elimination of the deadly virus in human patients as well.
Currently, HIV treatment is contingent upon a cocktail of antiretrovirals, and while these drugs can prevent the virus from replicating, it doesn’t serve as a real cure. Moreover, Temple University points out, “When antiretroviral therapy is interrupted, HIV replication rebounds, placing patients at risk for developing acquired immune deficiency syndrome, or AIDS.” But if doctors are able to fully remove the virus from human genomes altogether, a wholesale solution may finally be in sight.
In conducting their experiment, researchers first infected rats with HIV DNA, then engineered an ex vivo experiment to “cleave the integrated HIV-1 DNA in the host cell genome.” This would potentially excise the viral DNA fragment from the genome altogether. After two weeks, the Temple University team examined their subjects’ DNA, and found that the HIV DNA was no longer present, seemingly removed from every tissue, including the brain, heart, kidney, liver, lungs, spleen, and blood cells.
“The ability of the rAAV delivery system to enter many organs containing the HIV-1 genome and edit the viral DNA is an important indication that this strategy can also overcome viral reactivation from latently infected cells and potentially serve as a curative approach for patients with HIV,” said Dr. Khalili.
The next step for Khalili’s team will be to conduct a larger study on animals, but the team hopes that a clinical trial for human patients could come to fruition within the next several years.
EmoticonEmoticon