How Can Zinc Finger Proteins be used in Gene Therapy?
Zinc finger proteins can be engineered to induce double-stranded DNA breaks in specific DNA sequences, for example where a disease-causing bad gene is located - and then the cell's own repair machinery will go to work to correct the damage.
This offers a way of correcting genetic mutations without resorting to the traditional gene therapy approach which involves housing a desired gene in a vector and then sending it on its way into a body, with fingers crossed that it reaches its target and is then expressed in a way that is required.
How does it work?
The zinc finger DNA-binding domain is fused to the cleavage domain of the Fokl restriction endonuclease, and the complex is called a zinc finger nuclease which can induce the double-stranded breaks in specific DNA sequences.
The zinc finger portion identifies and binds to the DNA, and the cleavage domain chops the genetic sequence. The cellular repair machinery then makes good the damage, with a DNA template supplied by the scientists. The technology can also be used to stop the over expression of genes that may be contributing to a particular disease process.
According to the Zinc Finger Consortium the DNA-binding specificities of any DNA-binding domain can be engineered to target nearly all kinds of genetic sequences.