A New Field of Medicine
Genome editing makes permanent changes to the genetic code of a cell by correcting, disabling or modifying the DNA. Genome editing works by using enzymes called “engineered nucleases” that act like molecular scissors. By cutting DNA at a precise spot in the genome, DNA can be removed, added or replaced for therapeutic effect. The goal of genome editing is to provide a permanent therapeutic solution or cure for genetic diseases.
Using Zinc Finger Nucleases to Edit the Genome
Our zinc finger nuclease (ZFN) technology is based on a naturally occurring class of proteins called zinc finger DNA-binding proteins (ZFPs), which recognize and bind to specific sequences of DNA. We engineer ZFNs for precision, efficiency, and specificity, which we believe are the critical parameters for a therapeutic genome editing technology. The ZFNs make a double stranded break in the DNA in a precise location in the albumin gene, where the transgene is permanently integrated using the cell’s natural repair mechanism.
Advantages of ZFNs for designing genome editing therapeutics
- Precision: Ability to target any desired nucleotide in the human genome
- Efficiency: Level of editing at the desired target nucleotide or sequence
- Specificity: Editing the target nucleotide or sequence without editing anywhere else in the genome