Gibson assembly in human cells

CRISPR-based technologies are transforming gene therapy for severe genetic disorders, but developing a personalized genome-editing approach for every pathogenic mutation seems unfeasible. Conversely, replacing a defective DNA sequence with a functional one could enable the development of universal, mutation-agnostic gene therapies that could be accessible to patients. Although promising, achieving medium- to large-size genetic modifications remains challenging in therapeutically relevant cells. In our recently published manuscript in Nature,1 we describe prime assembly, a genome-editing modality that enables targeted DNA sequence assembly and integration in human cells (Figure 1).