MOST APPROACHES TO GENE therapy involve deleting or replacing faulty genes. But for some genetic disorders, repairs may be possible. That potential is described in the Apr. 28 issue of Proceedings of the National Academy of Sciences by researchers from the University of North Carolina and Bern University in Switzerland.
Messenger RNA molecules ferry information from DNA in a cell's nucleus to its outlying cytoplasm, where proteins are made. Malformed RNA is linked to a number of serious illnesses, including a hemoglobin deficiency called beta-thalassemia. This malady, common in the Middle East and Southeast Asia, can cause anemia and death in children.
Working with modified human cells, researchers were able to correct the problem with molecular bandages. The patches, known as "antisense" molecules, are mirror images of the sites producing faulty RNA. Thus, they fit perfectly over problem sites, blocking the production of defective RNA. In culture, treated cells responded with an increase in globin, a key protein in hemoglobin. The researchers expect to begin animal studies soon.