Prime editing can potentially repair the vast majority of known disease-causing human mutations, but the technology, first developed in 2019, has not yet been widely used in the body, or in vivo, to treat genetic disease. The only clinical application of prime editing that has been publicly announced uses the technology to edit cells outside the body before transplanting them back into the patient.

Now, scientists from the David Liu lab at the Broad Institute have addressed bottlenecks that previously impeded the use of prime editing in animals and human patients. In two studies published last month in Nature Biotechnology and one published today in Nature Nanotechnology, the team described multiple changes to key components of the prime editing system and also optimized prime editing for delivery with lipid nanoparticles, which deliver genetic medicines to tissues in the body and are already used in several approved therapeutics. These advances together increase the efficiency and potency of prime editing and improve its potency when delivered into the body, a key requirement for in vivo prime editing therapeutics.

"Collectively, these three papers improve the overall efficiency and clinical relevance of prime editing, which we hope will make the technique more useful both for research purposes and for therapeutic clinical applications," said Liu, who is also the Richard Merkin Professor and director of the Merkin Institute for Transformative Technologies in Healthcare at Broad, the Dudley Cabot Professor of the Natural Sciences in the Faculty of Arts and Sciences at Harvard University, and a Howard Hughes Medical Institute investigator.