Researchers Engineer Immune Cells to Resist HIV Infection

Researchers at the Stanford University School of Medicine have devised a way to engineer key cells of the immune system so that they remain resistant to infection with HIV, the virus that causes AIDS.

A new study describes the use of a sort of molecular scissors to cut and paste a series of HIV-resistant genes into T cells, which are specialized immune cells targeted by the AIDS virus. The genome editing was made in a gene that the virus uses to gain entry into the cell. By inactivating a receptor gene and inserting additional anti-HIV genes, the virus was blocked from entering the cells, preventing it form destroying the immune system, said Matthew Porteus, M.D., and an associate professor of pediatrics at Lucile Packard Children's Hospital.

Proteus, the study's principal investigator, explained:

"We inactivated one of the receptors that HIV uses to gain entry and added new genes to protect against HIV, so we have multiple layers of protection -- what we call stacking. We can use this strategy to make cells that are resistant to both major types of HIV."

Porteus said that the new approach is a form of tailored gene therapy, and could ultimately replace drug treatment. Patients currently have to take multiple medications on a daily basis in order to keep the virus in check and prevent the potential fatal infections brought by AIDS.

Because the work was done in a laboratory, clinical trials would still be needed in order to determine whether the approach would work as a therapy.

Sara Sawyer, Ph.D., an assistant professor of molecular genetics and microbiology at the University of Texas-Austin and a co-author of the study, gives more insight into the findings, explaining:

"Providing an infected person with resistant T cells would not cure their viral infection. However, it would provide them with a protected set of T cells that would ward off the immune collapse that typically gives rise to AIDS."

The study was published in the January 22 issue of Molecular Therapy.