Gene Therapy - The Future Is Here!

Medical Author: Frederick Hecht, MD, FAAP, FACMG

Gene therapy is the treatment of disease by replacing, altering, or supplementing a gene that is absent or abnormal and whose absence or abnormality is responsible for the disease. Gene therapy may use the genetic material, DNA, itself as the means of treatment.

DNA or deoxyribonucleic acid is the very long molecule that encodes the genetic information. A gene is a stretch of DNA required to make a functional product such as part or all of a protein. People have about 100,000 to 150,000 genes. During gene therapy, DNA that codes for specific genes is delivered to individual cells in the body.

Most, if not all, diseases have a genetic factor. The genetic factor can be wholly or partially responsible for the disease. For example, in disorders such as cystic fibrosis, hemophilia, and muscular dystrophy, changes in a gene directly result in the condition. In other conditions such as high cholesterol and high blood pressure, genetic and environmental factors interact to cause disease. Disorders associated with aging often involve the loss of gene activity in specific types of cells. Even infections can be related to genes. In fact, they have two sets of genetic determinants: the genes of the infective agent and the genes of the person with the infection.