Our research integrates chemistry and evolution to illuminate biology and enable next-generation therapeutics. Our major research interests include the engineering, evolution, and in vivo delivery of genome editing proteins such as base editors and prime editors to study and treat genetic diseases; the evolution of proteins with novel therapeutic potential using phage-assisted continuous evolution (PACE); and the discovery of bioactive synthetic small molecules and synthetic polymers using DNA-templated organic synthesis and DNA-encoded libraries.

Base editing—the first general method to perform precision gene editing without double-stranded breaks, and a Science 2017 Breakthrough of the Year finalist—as well as prime editing, PACE, and DNA-templated synthesis are four examples of technologies pioneered in his laboratory. These technologies are used by thousands of labs around the world and have enabled the study and treatment of genetic diseases.

At least 14 clinical trials using base editing or prime editing are already underway to treat diseases including leukemias, hypercholesterolemia, sickle-cell disease, and beta-thalassemia, and chronic granulomatous disease. The first clinical benefits of both ex vivo and in vivo base editing—including young T-cell leukemia patients whose lives were saved by base editing and sickle-cell disease patients who are now symptom-free and medication-free—have already been reported.