Definition for View source for Germline therapy

Gene therapy is a novel approach to treating diseases based on modifying the expression of a person’s genes towards a therapeutic goal. Biotechnology has advanced to the point where scientists can alter the DNA; the genetic code that defines us. There are essentially two forms of gene therapy, Somatic Gene Therapy and Germline Therapy. Somatic gene therapy involves the manipulation of gene expression in cells that will affect the patient but will not be inherited.

Germline gene therapy involves the modification of germ cells (gametes) that will pass the change on to the next generation. With germline therapy genes could be corrected in the egg or the sperm that is being used to conceive. The child that results would be spared certain genetic problems that might otherwise have occurred. Because every cell descends from the fertilized egg, every cell in the offspring would possess the transplanted gene. This would be a far more effective way of transferring genes than the ones presently used in somatic cell therapies, where genes into the cells of children or adults usually enter only a small portion of the person’s cells and eventually stop functioning.

Some 2000 to 3000 diseases have been determined to be genetic or inherited. Up to two percent of newborns in the U.S. have a genetic disorder. The most common of these, cystic fibrosis affects 30,000 Americans and is found in 1700-2000 newborns each year. Adenosine Deaminase Deficiency (ADA) is a severe immunodeficiency disorder that affects 40-50 people worldwide. <br>