Introductory Paragraph PART A

You start to sniffle, cough, and get a sore throat. You know that you’re about to endure a few weeks of the common cold. While this may be an annoying inconvenience, you know that it will eventually go away and you will return to health. Now, imagine being born with an illness that you will have your entire life—but it is much worse than the common cold. What would it be like to have to go through your entire life afflicted with an illness like Huntington’s disease, hemophilia, severe combined immune deficiency (SCID), or cystic fibrosis? Not only do these diseases severely impair the health of an individual, they and many others like them are incurable. A doctor cannot prescribe an antibiotic to treat these diseases, because they are caused by a defective genetic code, not a pathogen. It has only been within the last hundred years that scientists have discovered the cause of genetic diseases which were a profound medical mystery up until the discovery of genes and DNA as the genetic blueprint. Modern science has made certain therapies available for some of these diseases so that individuals with Type I diabetes, for example, can receive insulin injections, and hemophilia patients can receive supplements of the clotting factor that their body does not produce. However, many of these genetic diseases, such as SCID, have no treatment to supplement the deficiencies of a faulty genetic code. Children born with SCID are confined to a germ-free “bubble” their entire lives unless they are lucky enough to get a bone marrow transplant that their body does not reject. Being diagnosed with a genetic disease is extremely sad because there is usually little to no hope for a cure or even a treatment.

However, within the last couple decades researchers have discovered a new way to treat genetic diseases. Instead of just providing supplements and making up for what the body doesn’t produce, why not alter the human genome and “fix” the genes? Gene therapy, as this new treatment is called, is much more complicated than most people, and even most scientists, comprehend, but it is with reach. In 1998, for example, ten SCID patients were treated using gene therapy and all but one were able to live normal lives thanks to this new alternative treatment. However, anytime a scientist wants to mess with the human genome, there is ethical controversy. Though gene therapy research generally does not involve the use of embryonic stem cells, bioethicists are concerned that gene therapy will lead to the standardization of eugenic practices, especially as regards fetal gene therapy. Also, many of the clinical trials associated with gene therapy have failed. Mistakes in gene therapy research protocol have resulted in tight restrictions on the progress of research by the federal government. With more research and safer clinical trials, gene therapy could eventually become an important treatment for genetic diseases. Currently, researchers are also using gene therapy to treat certain types of cancer and preliminary research is now underway to test gene therapy as a treatment for AIDS. Through controversy surrounds the study of gene therapy, this life-saving science could be more extensively researched, funded, and eventually commercialized so that people born with serious genetic diseases can have hope for a healthy, productive life.