Science

Researcher Sees Path 'Towards a Cure' for HIV With Gene Editing

Jul 5, 2019

A groundbreaking new study reports that researchers were able to use gene therapy to eradicate HIV in mice. Now the team is considering how to turn that into a life-saving cure for humans.

"We actually have the tools now to go into the genome of a live animal — and inevitably into a person — and clip out, or excise out, the infectious HIV, in a means, or a pathway, towards a cure," said Howard Gendelman, a professor of internal medicine and infectious diseases at the University of Nebraska Medical Center, during an interview with Cheddar on Friday.

The treatment, which was discovered by teams at the University of Nebraska Medical Center and Temple University, works by "dampening down" the HIV virus through an antiretroviral therapy called LASER ART, and then using the gene-editing tool CRISPR-Cas9 — essentially a genetic "scissor" — to remove genes impacted by the virus.

The method led to a "total elimination" of the virus in nine of 23 animals, Gendelman said. It is the first time the virus has been completely eradicated from an animal, according to a UNMC press release.

But Gendelman cautions that applying the technique to humans requires both improving the technology for locating DNA impacted by the virus, and ensuring that the treatment doesn't damage other vital portions of the human genome.

"Every single HIV DNA that's integrated into the genome of a live host has to be excised. So we must develop even better technologies than we've developed in mice to discover where that latent virus is," explained Gendelman. "That's a major part of the research efforts ongoing now."

"We're manipulating the human genome. We're treating an HIV infection as if it was a genetic disorder. We're going into the genome and changing the structure, essentially using the HIV as a bad gene and cutting it out," he added. "So if we do that, we have to understand that there's a possibility that other genes can be affected."

More than 70 million people are reported to have been infected with HIV, according to the World Health Organization. The U.N. agency estimated that in 2017, more than 35 million people were living with the virus.