Gene Therapy
SCI115 Introduction to Biology
Professor Cassie Prisco
November 28, 2014

Gene therapy is a rapidly growing field of medicine in which genes are introduced into the body to treat diseases. It is the name applied to the treatment of inherited diseases by corrective genetic engineering of the dysfunctional genes. It is part of a broader field called genetic medicine, which involves the screening, diagnosis, prevention and treatment of hereditary conditions in humans. The results of genetic screening can pinpoint a potential problem to which gene therapy can sometimes offer a solution.
Genes represent the genetic material that organisms pass on from generation to generation. Therefore, genes are responsible for controlling hereditary traits and provide the basic biological code or blueprint for living organisms. Genes produce protein such as hair and skin as well as proteins that are important for the proper functioning of organs. Mutated or defective genes often cause disease. The purpose of gene therapy is to replace a defective gene with a normal copy of the same gene in attempt to restore function.
Initially conceived as an approach for treating inherited diseases, like cystic fibrosis and Huntington's disease, the scope of potential gene therapies has grown to include treatments for cancers, arthritis, and infectious diseases. Although gene therapy testing in humans has rapidly advanced, in general, the field of gene therapy has proven to be problematic and complicated by a variety of ethical issues. For example, some scientists are concerned that the integrating genes into the human genome may cause disease (Bio Technology). There has been evidence that randomly integrating corrected genes might disrupt other genes in the genome and if the disrupted gene is a tumor suppressor gene, cancer may develop. Others fear that germ-line gene therapy may be used to control human development in ways not connected with disease, like intelligence or appearance.
The biology of human gene therapy is very complex, and there are many techniques that still need to be developed and diseases that need to be understood more fully before gene therapy can be used appropriately. Because gene therapy involves making changes to the body’s genetic setup, it raises many unique ethical concerns (Starr, Evers, & Starr 2013). Scientific and ethical discussions about gene therapy began many years ago, but it was not until 1990 that the first approved human gene therapy clinical trial was initiated (Gene Therapy). This clinical was considered successful because it greatly improved the health and well-being of the few individuals who were treated during the trial. The success of the therapy was tentative, because along with the gene therapy the patients also continued receiving their traditional drug therapy. This made it difficult to determine the true effectiveness of the gene therapy on its own, as distinct from the effects of the more traditional therapy.
Measuring the success of treatment is just one challenge of gene therapy. Research is fraught with practical and ethical challenges. As with clinical trials for drugs, the purpose of human gene therapy clinical trials is to determine if the therapy is safe, what dose is effective, how the therapy should be administered, and if the therapy works (Bio Technology). Diseases are chosen for research based on the severity of the disorder. Imagine you or your child has a serious condition for which no other treatment is available. How objective would your decision be about participating in the research?
How do researchers determine which disorders or traits warrant gene therapy? Unfortunately, the distinction between gene therapy for disease genes and gene therapy to enhance desired traits, such as height or eye color, is not clear-cut. No one would argue that diseases that cause suffering, disability, and, potentially, death are good candidates for gene therapy. However, there is a fine line between what is considered a "disease" (such as the dwarfism disorder achondroplasia) and what is considered a "trait" in an otherwise healthy individual (such as short stature). Even though gene therapy for the correction of potentially socially unacceptable traits, or the enhancement of desirable ones, may improve the quality of life for an individual, some ethicists fear gene therapy for trait enhancement could negatively impact what society considers "normal" and thus promote increased discrimination toward those with the "undesirable" traits. As the function of many genes continue to be discovered, it may become increasingly difficult to define which gene traits are considered to be diseases versus those that should be classified as physical, mental, or psychological traits.
To date, acceptable gene therapy clinical trials involve somatic cell therapies using genes that cause diseases. However, many ethicists worry that, as the feasibility of germ line gene therapy improves and more genes causing different traits are discovered, there could be a "slippery slope" effect in regard to which genes are used in future gene therapy experiments. Specifically, it is feared that the acceptance of germ line gene therapy could lead to the acceptance of gene therapy for genetic enhancement. Public debate about the issues revolving around germ line gene therapy and gene therapy for trait enhancement must continue as science advances to fully appreciate the appropriateness of these newer therapies and to lead to ethical guidelines for advances in gene therapy research (Ethical and Social Issues in Gene Therapy). Major participants in the public debate have come from the fields of biology, government, law, medicine, philosophy, politics, and religion, each bringing different views to the discussion.
Diseases like cystic fibrosis or diabetes could potentially be treated with somatic cell gene therapy, but parents may, for good reason, want to relieve their children of the necessity of undergoing such treatment or of the responsibility of transmitting unwanted harmful genes to their own children. Moreover, it seems far more efficient to cure a genetic disease with a single treatment that will prevent its appearance in future generations, than to treat the disease each time it appears, generation after generation. From a public health perspective, efficient prevention of disease is an important social value, as is conservation of future health care resources (Ethical and Social Issues in Gene Therapy). Germ-line gene therapy may lead to both of these outcomes.
To me there are benefits to the modern technology of gene therapy. It has the potential to cure horrible and life-threatening genetic diseases. Less people would die of diseases that they had no control over getting. For instance, type 1 Diabetes, an inherited disease, could be genetically changed and then life wouldn’t be as restrained as it is now. Gene therapy also has the potential to save millions of lives. If all of the genetic diseases were changed and cured in one generation as a whole, the generations to come would be cured of the diseases. Life for everybody would be drastically changed. Gene therapy has the ability to create an easier life for people world-wide. With less bodily problems, life can be lived in an easier and more relaxed manner. That would give everyone one less thing to worry about. Even though gene therapy has the potential to be a wonderful discovery, it also has many issues that need to be sorted out. There are millions of questions that must be answered before gene therapy can go any farther. Are disabilities diseases and do they need to be cured/ prevented? People have different ideas on what disabilities are. Since it depends on ethics, different people will have different thoughts of what a disability is and what it is not. This brings the question, what is a disability/ disorder, and who gets to decide. It will be a matter of opinion and bring about controversial comments from everyone. Gene therapy is a very expensive procedure. Who is going to pay for this and who will it be available to? Genetic disorders come to everybody: rich or poor. And for those who have wealth, would they be able to get this procedure done to change their hair color, eye color, height? These are all burning questions that will not allow gene therapy to go any farther unless they are answered. All in all, gene therapy is a miraculous new discovery that could change mankind for better or worse. If the questions are answered and answered ethically, then this will be a wonderful modern possibility.

References
Bio Technology. Retrieved from http://biotech- genetics.blogspot.com/2008/12/biological-basis- of-gene-therapy.html
Ethical and Social Issues in Gene Therapy. Retrieved from http://www.genetherapynet.com/ethical-and-social-issues-in-gene-therapy.html
Gene Therapy - The Biological Basis of Gene Therapy, Viral Vectors, The History Of Gene Therapy, Diseases Targeted For Treatment By Gene Therapy. Retrieved from http://science.jrank.org/pages/2964/Gene-Therapy.html
Starr, C., Evers, C. A., & Starr, L. (2013). Biology today and tomorrow without physiology (4th ed.). Belmont, CA: Brooks / Cole Publishing Co. (Cengage Learning)