Scientists have removed HIV from the DNA of mice modified so their immune systems would better mimic those of humans, bringing researchers a step closer to finding a cure for people living with the virus.
The news was delivered in a study published this week, and credited to more than 30 scientists from Temple University and the University of Nebraska Medical Center. Using a combination of sequential long-acting slow-effective release antiviral therapy (LASER ART) and a gene editing tool called CRISPR, researchers eliminated HIV in nine of 23 mice.
Clinical trials for the gene-editing component of the cure could begin next year, pending approval from the Food and Drug Administration, Kamel Khalili, one of the study’s senior investigators, told The Washington Post.
Khalili said he and his team had been waiting for a tool like CRISPR and that “the outcome was amazing,” while also cautioning it remains to be seen if the results can be replicated in humans.
“The big message of this work is that it takes both CRISPR-Cas9 and virus suppression through a method such as LASER ART, administered together, to produce a cure for HIV infection,” Khalili said in a statement. “We now have a clear path to move ahead to trials in non-human primates and possibly clinical trials in human patients within the year.”
Howard Gendelman, chairman of UNMC’s pharmacology and experimental neuroscience department and study author, told USA Today the process “only is successful if we get rid of every virus in the body.”
“We had to be highly efficient and getting a third of these animals cured is amazing considering what we were up against,” he said.
“We’re working on this day and night and we hope it’ll be sooner than later, but we have some obstacles to overcome,” he added. “There’s a tremendous amount of effort to move this technology forward.”
While three individuals have been successfully rid of the virus, scientists have said it could return in those cases, leading many to prefer the term “long-term remission” as opposed to “cured.” The treatment those men received—bone marrow transplants from donors immune to HIV, implemented in each case as a final effort to cure each man’s cancer—are painful, risky, and can bring serious side effects, making them less than ideal for widespread application.