The New Genetic Medicines; December 1994; Scientific American Magazine; by Cohen, Hogan; 6 Page(s)

To someone interested in curing a disease, nothing would be more gratifying than discovering a "magic bullet"--a drug able to reverse the illness without producing side effects. For most of the 20th century, researchers hoping to find magic bullets thought in terms of agents able to combine with the active sites of proteins that contribute to disease. By filling these active sites, which differ from one protein to another, the drugs would presumably inhibit the activity of the targeted proteins but would not interfere with needed ones.

Work on proteins is ongoing. Recently, however, a number of investigators have turned their attention to an exciting new class of targets. Following what are known as the triplex and antisense strategies, those researchers are attempting to design drugs that will bind to selected sites on the nucleic acids (DNA and RNA) that direct the synthesis of disease-related proteins.