Prime assembly enables universal gene therapies by correcting multiple mutations at once

A new genome engineering method called prime assembly, described today in Nature, can stitch long DNA fragments into precise genome locations, potentially enabling universal gene therapies that correct multiple mutations at once. Prime assembly builds on prime editing, allowing large, permanent DNA inserts without double-strand breaks or untargeted delivery, reducing toxicity and off-target risks. It also works in nondividing cells, unlike many current methods that are limited to dividing cells. The team plans to refine delivery to disease-relevant cells in vivo, such as hematopoietic stem cells, aiming for mutation-agnostic treatments for inherited diseases with unmet clinical needs.