When biochemist Jennifer Doudna and her research partner, Emmanuelle Charpentier, published a paper in Science 12 years ago, they had a hunch that their findings would transform how genomics is used in medicine. The paper outlined a method they’d developed for editing DNA that used an RNA-based system known as CRISPR-Cas9. The approach was more efficient and precise than any prior gene-editing technology.
By the time Doudna and Charpentier were awarded the Nobel Prize in chemistry in 2020, CRISPR (short for “clustered regularly interspaced short palindromic repeats”) had engendered a biotechnology revolution. Today, researchers around the world are employing CRISPR to develop diagnostics for the Zika virus, disease models for Alzheimer’s, and therapeutics capable of attacking cancer cells. What started as a “cut, copy, and paste” gene-editing technique has expanded into a far broader toolkit for human health.
Last year, the technology began having real-world effects when the FDA approved the first CRISPR-based gene therapies for sickle cell disease. “It’s extraordinary talking to some of the patients,” Doudna says, who are finding the treatment “completely life-changing.”