The Power of Gene Therapy
Each day as our technology advances, we become aware of new diseases and disorders. We also find effective ways to alleviate a number of these problems. In the last decade, gene therapy has been found to treat a portion of life-threatening illnesses such as Cystic Fibrosis, Severe Combined Immune Deficiency (SCID), and Alzheimer's disease.
Cystic Fibrosis (CF) is a genetic disease that affects the exogenous secreting glands of the body and generates thickened secretions, therefore blocking certain functions of the lungs and pancreas. Approximately thirty thousand people in the U.S. and many more throughout the world have cystic fibrosis. It is inherited in an autosomal recessive fashion; heterozygotes who carry one normal CF allele and one mutant allele are only carriers, whereas the child of two carriers has a one in four chance of inheriting a mutation from each parent and becoming stricken with CF. By using gene therapy, doctors have begun to treat the disease by transferring a critical gene into the patients' airways. The idea is, if the gene can enter the cells lining the lungs, it should allow the cells to produce the protein that CF patients cannot make themselves. Results show that after undergoing this gene therapy procedure, patients have been able to produce this critical protein, but only in minute quantities. Although this method has not yet been perfected, there is a good chance that it will soon be a widely used and effective treatment that will save the lives of many CF patients.
Gene therapy has also been found efficient in treating those with Severe Combined Immune Deficiency (SCID). SCID is a genetic disorder that afflicts one in every one hundred thousand babies. Several genes are at fault in this disorder, and babies are eventually left with no T cells. A lack of T cells means a lack of an enzyme called adenosine deaminase (ADA) that breaks down chemicals that are poisonous to the body. Without T cells and ADA, one basically has no immune system. To correct these genes, stem cells (precursors of T cells) are taken from the blood of a newborn's umbilical cord. A retrovirus given a normal ADA gene is added to the stem cells. Next, the virus inserts its gene information into the cells. The new cells are then returned to the baby. Until the babies can make enough ADA on their own, they are given...