Date: 06/05/2025

by Isaac P. Witte, George D. Lampe, Simon Eitzinger, Shannon M. Miller, Kiara N. Berríos, Amber N. McElroy, Rebeca T. King, Olivia G. Stringham, Diego R. Gelsinger, Phuc Leo H. Vo, Albert T. Chen, Jakub Tolar, Mark J. Osborn, Samuel H. Sternberg, and David R. Liu

Scientists are working on ways to insert large pieces of DNA into specific spots in the human genome. This could eventually allow doctors to treat many different genetic diseases with a single type of drug. There's a natural tool called CAST that can insert big chunks of DNA using a guide (like GPS), but until now, it hasn't worked well in human cells.

To fix that, researchers led by Witte created a system that helps improve CAST's performance over time. The result is a much better version of CAST—over 200 times more effective in human cells. This upgraded tool can now insert genes into many important spots in human DNA across different types of cells, opening up powerful new possibilities for treating diseases and editing human genes. https://www.science.org/doi/10.1126/science.adt5199