Scientists at the Stanford University School of Medicine have developed a technique to genetically engineer key immune system cells and make them resistant to HIV, the virus that causes AIDS, according to a new study.
HIV patients could ultimately be offered an alternative to taking a lifetime of multiple medications if the new approach, described by researchers as “genome editing” proves successful in human subjects.
“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,” said Matthew Porteus, MD, an associate professor of pediatrics at Stanford and the study’s principal investigator. “We can use this strategy to make cells that are resistant to both major types of HIV.”
Research is still in the early stages and will need to be tested more thoroughly in T-cells taken directly from AIDS patients, then in animals. Stanford scientists hope to begin clinical trials within three to five years — trials which are needed to determine whether the technique would work as a therapy.
Dr. Porteus is convinced that this new approach is an important step forward in developing a gene therapy for HIV and could ultimately replace drug cocktail treatments which are known to have adverse side-effects.
“To develop novel therapies you have to be an optimist,” said Dr. Porteus. “The findings in this study are a proof of concept; we’ve proven this could work. I’m very excited about what’s happened already.”
The study published in the Jan. 22 issue of the journal Molecular Therapy, was funded by the Foundation for AIDS Research.