The advantages of ZFN-mediated genome editing
We believe that our ZFN technology has advantages over other genome editing technologies and traditional gene therapy approaches.
The ability to place a gene-sized segment of DNA into a pre-determined location in the genome, with singular specificity, eliminates insertional mutagenesis concerns associated with traditional lentiviral gene replacement approaches and broadens the range of mutations of a gene that can be corrected in a single step.
ZFNs provide the best platform for therapeutic genome editing because they enable:
- Precision: Exquisite targeting precision -- high design density means we can place ZFNs at virtually any sequence in the genome
- Specificity: We can quickly engineer ZFNs to be highly specific -- engineered to engage a target site with no meaningful off-target effects
- Efficiency: We can quickly engineer ZFNs to be extremely efficient -- to cut DNA with high on-target efficiency (>90% for initial lead design screens), while retaining excellent specificity