Human Gene Therapy

Abstract

There are more than 4,000 known inherited disorders. The majority of them have minimal effects, but a few causes physical and mental abnormalities that may be life threatening. Genetic diseases that are candidates for gene therapies include severe combined immunodeficiency, thalassaemia, and cystic fibrosis. Since these genetic diseases are each caused by a single defective gene, one potential treatment is to introduce a normal functional copy of the appropriate gene into the cell tissue that is affected. In effect, the normal (therapeutic) gene augments the defective gene in the patient. Gene therapy is not restricted to only treating genetic disorders. The general technology of transferring genetic materials into a patient is also applied to diseases such as cancer, AIDS, and cardiovascular diseases. Many of the approved clinical trials on gene therapy are for the treatment of diseases other than genetic disorders.