Gene therapy

Gene therapy, a technique for correcting defective genes, is offering new and exciting possibilities for curing and treating several genetic disorders. Instead of treating the symptoms of a disorder, gene therapy alters the genetic makeup of certain cells. Researchers may use one of several approaches for correcting faulty genes.

The revolutionary approach of gene therapy was first used in 1990, and the original goal was to substitute a healthy gene for a defective one or to repair a faulty gene, thereby eliminating symptoms of disease. But researchers have moved beyond inherited genetic disorders to treat other kinds of diseases. Today, almost 75 percent of all clinical trials involving gene therapy are aimed at treatments for cancer and acquired immunodeficiency syndrome.