A new method, called “prime editing,” could, in principle, correct around 89 percent of the mutations that cause inherited human disease.
Anzalone’s prime editor is a little different. Its enzyme is actually two that have been fused together—a molecule that acts like a scalpel combined with something called a reverse transcriptase, which converts RNA into DNA. His RNA guide is a little different too: It not only finds the DNA in need of fixing, but also carries a copy of the edit to be made.
The bigger problem, according to folks like Burgio, is that prime editors are huge, in molecular terms. They’re so big that theyinto the viruses researchers typically use to shuttle editing components into cells. These colossi might even clog a microinjection needle, making it difficult to deliver into mouse embryos. That, says Burgio, could make prime editing a lot less practical than existing techniques.
But that’s not stopping Liu from moving forward with plans to bring prime editing to patients. In September, he cofounded a company called Prime Medicine that has licensed the technology from the Broad Institute to develop treatments for genetic disease. Liu’s base editing company, Beam Therapeutics, has also been granted a sublicense for certain fields.
As for Anzalone, he’s just astonished at how fast something can move from a flash of curiosity to a working molecular machine in the world of Crispr. “There are things that we can do now that seemed impossible when I began graduate and medical school,” he says. That means there's plenty of fodder for more evening strolls, but this time in Cambridge, Massachusetts, instead of Manhattan.
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