Targeted insertion for human genome engineering

While we have multiple technologies for small allele genome editing, we do not have equally robust technologies for large alleles. We have developed a precise gene delivery tool combining the precision of programmable DNA binding domains such as CRISPR (find module), and the insertion efficiency of transposable elements (cut-and-paste module) such as piggybac variants. We have developed a system for highly precise programmable insertion in mammalian genomes. We have demonstrated efficient delivery of small and large payloads to 293T, Jurkatt cells and in vivo mouse liver.