Precise DNA insertion method enables long gene edits in living cells
A new genome engineering method called prime assembly enables long DNA fragments to be inserted precisely into living cells, potentially enabling universal gene therapies for many patients. The findings were published today in Nature. Prime assembly builds on prime editing, allowing large, gene-sized DNA pieces to be stitched into targeted genome positions in a single step. It avoids off-target effects, toxicity, and double-strand breaks, and works in nondividing cells, unlike other methods. The team aims to improve delivery of components to human cells in vivo, such as hematopoietic stem cells for blood disorders. They hope to develop mutation-agnostic therapies for inherited diseases with unmet clinical need.