Cut, Paste, Cure

"This technology almost immediately revolutionized the way people were doing basic science," said David Taylor, an associate professor of molecular biosciences at UT, who was previously a postdoctoral researcher in the lab of a scientist who won the 2020 Nobel Prize in chemistry for her CRISPR research. Scientists once spent years building objects to target specific genes, Taylor observed. He likened the effort to making a new computer each time one wanted to log onto a different website. CRISPR, by contrast, offers simplicity on a par with downloading an app.

"Before, we had clunky ways to study genes," he said. "With CRISPR, scientists suddenly had a tool to study any gene they wanted to target for any disease or process inside a cell. That's what has made it such a game-changing technology."

CRISPR-Cas9 is a surprisingly cheap editor that needs only a few days or weeks to set up and run, at least in simple organisms. Targeting specific cells, particularly in living animals, remains more challenging, but the technology has already produced mosquitos that don't transmit malaria and fashioned chickens and mice that are impervious to certain genetic disorders. It has allowed pig organs to be adapted for transplant into humans, corrected a genetic version of blindness, helped edit patients' own cells to treat sickle cell disease and found its way into a gene therapy, used last year for the first time to treat a living patient - thereby curing a 9-month-old baby of a rare, fatal genetic disease.

"This has been one of the fastest bench-to-bedside technologies ever," marveled Ilya Finkelstein, another associate professor who studies CRISPR systems in UT's Department of Molecular Biosciences.

"This has been one of the fastest bench-to-bedside technologies ever."

The ability to disrupt a single gene helps scientists see how it affects the entire organism, much as changing a single word or phrase in a poem alters its entire effect. Since CRISPR came online, molecular biologists have built whole libraries to document the relationships between genes and what they do. Nonetheless, researchers say, CRISPR technologies' full potential will be even greater when scientists learn more about the basic grammar of their new editor.