Penn Researchers Getting Close to ‘Functional Cure’ for HIV?

A new study from Penn scientists uses the new technique of gene editing. It may help fight HIV.

A study in this week’s New England Journal of Medicine has exciting news: Penn researchers may be close to finding a ‘functional cure’ for HIV.

What’s even cooler is how the doctors at Penn did this: They edited a gene — “snipped” it out, per the Inquirer‘s Marie McCullough — to make patients partially resistant to HIV.

The researchers’ editing tool, developed by Sangamo BioSciences of Richmond, Calif., was made of natural proteins that recognize specific DNA sequences. These “zinc finger nucleases” can be used like molecular scissors to introduce intentional genetic mutations.

“The ability to edit the human genome has been a prayer ever since we first understood that genes control biology,” said Sangamo CEO Edward Lanphier, who founded the company in 1995. “But we’ve moved beyond the concept of gene replacement, which was the idea behind gene therapy. Gene editing is much safer and more effective.”

And you wanted to live in a world without zinc. Sangamo is also working on gene editing cures for hemophilia, Huntington’s disease, and sickle-cell anemia.

Four patients who suspended HIV drugs after gene editing saw their blood levels of HIV fall. We’re still ways away from a cure and, especially since gene editing is in its infancy, this may not work. But, as the Inky describes it, this is a “game changer.” For once that cliche seems accurate.