Gene Therapy

Gene therapy is the introduction of a normal gene into an individual’s genome in order to repair a mutation that causes a genetic disease. When a normal gene is inserted into a mutant nucleus, it most likely will integrate into a chromosomal site different from the defective allele; although this may repair the mutation, a new mutation may result if the normal gene integrates into another functional gene. If the normal gene replaces the mutant allele, there is a chance that the transformed cells will proliferate and produce enough normal gene product for the entire body to be restored to the undiseased phenotype. So far, human gene therapy has been attempted only on somatic (body) cells for diseases such as cancer and severe combined immunodeficiency syndrome (SCIDS). Somatic cells cured by gene therapy may reverse the symptoms of disease in the treated individual, but the modification is not passed on to the next generation. Germinal gene therapy aims to place corrected cells inside the germ line (e.g., cells of the ovary or testis). If this is achieved, these cells will undergo meiosis and provide a normal gametic contribution to the next generation. Germinal gene therapy has been achieved experimentally in animals but not in humans.