If it can be developed to the stage where individual genes can be targeted accurately, it may offer a wide range of therapeutic benefits. Regulating genes artificially could hold the key to several genetically based and viral diseases. Cells regulate their own genes all the time using proteins that bind to specific stretches of DNA, thereby diminishing or blocking the conversion of DNA-encoded information into the protein it represents.
Some genetic diseases result from the inappropriate activation of genes.
This can cause a cell to produce a protein in the wrong amount or at the wrong time. So researchers are attempting to devise synthetic molecules that, like natural DNA-binding proteins, can attach themselves to particular faulty or unwanted genes and inactivate them. Artificial DNA-binding molecules have been created that will bind only to stretches of DNA encoding a particular message. But it remains hard for these molecules to distinguish between very similar messages, so they are not guaranteed to stick to the right gene. For a DNA-binding drug to inactivate the wrong gene could be disastrous.