MIT Study Suggests Gene Therapy Could Be Used for HIV Treatment

It identified five genes that could be targeted therapeutically.

Broad Institute

Broad Institute photo by Kelly Davidson Photography

Researchers from the Ragon Institute and the Broad Institute may have found an innovative potential strategy for eradicating HIV.

The team used CRISPR-Cas9, a genome editing tool, to identify genes that HIV needs to survive, but that aren’t essential to human health.

“Developing new drugs to target human genes required for HIV infection is a promising approach to HIV therapy, with potentially fewer opportunities for the development of resistance,” Ryan Park, co-lead author of report and a researcher at the Ragon and Broad Institutes, said in a statement. This distinction is important, because current HIV medications mostly target proteins—but since HIV mutates, drug-resistant strains often develop.

Only nine genes make up HIV, while humans have more than 19,000 in all. Like other viruses, HIV poaches existing human genes to replicate and spread. Targeting host genes that are essential to HIV but unessential to humans, then, could be a viable therapeutic option.

Using CRISPR, the research team found five such genes, including two previously identified proteins that HIV needs to enter T cells, the primary targets of HIV infection. The other three had never before been flagged for potential HIV therapy. When those five genes were inactivated, they seemed to curb HIV infection without otherwise affecting the individual’s cellular health.

While gene therapy is still in its infancy and a costly potential therapy, results like these are promising.

“In addition to our specific findings related to the HIV infection process, our study demonstrates how CRISPR-based screens can be applied to identify host factors critical to the survival of other viral pathogens but dispensable for host cell viability,” co-lead author Tim Wang said in the statement. “Broad application of this method should pinpoint a novel class of potential therapeutic targets that have previously been underexplored for the treatment of infectious disease.”