A gentler gene edit repairs an immune defect in a patient's own cells

At Aarhus University in Denmark, Jonas Holst Wolff and Jacob Giehm Mikkelsen used prime editing — a precise form of gene editing that does not slice DNA in two — to repair the defect behind GATA2 deficiency directly in a patient's own blood stem cells. Published in Science Translational Medicine, the work brings treatments that fix the cause of genetic immune disorders, rather than only their symptoms, a step closer.

SDG 3 · Good health and well-being — Mechanism: tech innovation