Gene Therapy

Cystal Schilling

Copyright 1998


Imagine yourself being a parent of a young child suffering from a horrible disease in which you now know to be genetically inherited. This disease is caused from the genes that you and your partner passed on to your child. Now lets say, you can treat this disease or may even be able to cure your child of this disease by altering, modifying, or replacing the genes that cause this disease through a process called gene therapy. Would you do it? I think most of us would answer yes.

The option of being able to help our child several years ago may not have seemed possible. Genetic research has made giant leaps over the past decade and now the idea of gene therapy is reality. Through gene therapy it is possible to treat and elevate genetic diseases and mutations. We now have the knowledge and the technology to treat ourselves , our children, and the future generations.

In order to make an educated decision, one must be aware of gene therapy itself. When making such a decision of whether to use gene therapy or not it would be beneficial to understand the technical aspects of gene therapy. It is also important to understand how gene therapy treats an individual, what it can be used to treat, and the previous trials and successes of the use of gene therapy. Or a person may find it worthwhile to know how the public perceives gene therapy and the overall consensus on its usage. After obtaining all of this knowledge, the decision of whether or not to use gene therapy should be able to be made confidently.

Gene Therapy and the Technical Aspects

Gene therapy is a means of treating diseases based on modifying the expression of a person's genes towards the therapeutic goal. Gene therapy could be used in treating lethal debilitating diseases and also may have the potential for preventing diseases (Wilson, 10/20/97). The notion of practicing gene therapy to modify the expression of genes in ways that would be beneficial to ourselves stems from the understanding of the genetic basis of human disease.

The goal of gene therapy is to correct the mutations that have occurred within the DNA of our living cells. In simple terms DNA is the genetic material that contains the genes, nucleotide pairs, codons and genomes. In order to, "treat the individual" or correct the mistake, the identification and location of the gene(s) causing the problem must be determined.

A child inherits genes from his and her parents. These genes may cause disease or abnormalities. Each individual contains approximately 150,000 separate genes (Wilson, 10/20/97). These genes help define each person as an individual. Some of these genes we have inherited are referred to as defective genes. Each of us carries around six defective genes, but most of us are unaware of this unless someone in our family suffers from a genetic disease (Biotech Applied, 1990). Most of us don't suffer any harmful effects from these defective genes because we carry two copies of nearly all genes, one from our mother and one from our father. The exception being the genes found on the male sex chromosomes. Males having only one X and one Y chromosome, carry only one copy of the genes on each of the chromosomes. Usually one gene is sufficient to avoid symptoms of most inherited diseases. In cases where the harmful gene is recessive, we only will develop the disease if we inherit two copies of the same recessive gene. In cases of dominant genes, one gene alone can produce the disease (Biotech Applied, 1990).

Gene therapy is based on correcting the disease at its root, the root of the disease begins at the abnormal genes. The abnormal gene(s) which could be said to be faulty or may not even be present is replaced by a working gene(s). To be able to replace the abnormal gene(s), a means of delivery has to be available that is specific, efficient, and safe. One method developed that has been used in several trials is delivery of the gene through means of a vector. The vector encapsulates the desired genes for delivery to the cells. Many vectors used are attenuated or modified versions of viruses. The virus is modified by removing the disease causing components of the virus and inserting the desired genes that will be therapeutic to the patient. The modified virus cannot replicate in the patient but can efficiently deliver the desired genetic material. Another way of delivery is based on synthetic vectors in which complexes of DNA, lipids, or proteins are formed in particles capable of efficiently transferring genes (Wilson, 10/20/97).

There are two forms of gene therapy which potentially could be used, Somatic gene therapy and Germline gene therapy. Somatic gene therapy involves the manipulation of gene expression in cells that will be corrective to the patient but not inherited in the next generation. Germline therapy involves the genetic modification of germ cells that will pass the change on to the future generations (Wilson, 10/20/97).

Through gene therapy many individuals suffering from several different diseases potentially could be treated and relieved from their suffering that these diseases cause. Gene therapy gives promise in treating individuals living with such diseases as PKU, cardiovascular disease, ADA, AIDS, and many more (Wilson, 10/20/97). The first use of gene therapy, performed in September of 1990, was on a young girl suffering from ADA, a rare genetic immunodeficiency disease caused by the lack of the enzyme, adenosine deaminase. This disease was also the disease of the infamous "bubble boy." The young girl was treated every couple of months with the infusion of the ADA gene corrected cells through a viral vector. The results were positive. The young girl was able to attend school and participate in activities as a normal child within one year of the initial treatment (Grace, Jan-Feb 1998).

Ethical Issues: The Public Debate

Gene therapy could make it possible to engineer the genetic blueprints of our own species and begin to control the future of our own evolution (Rifkin, May-June 1998). The possibilities of gene therapy are enormous. What we can accomplish using gene therapy can be life saving and life creating. Gene therapy, as stated before, can treat diseases, and correct genetic mistakes in the individual and in future generations. Another use can be "creating and designing " individuals. These advances bring up many ethical issues for both researchers and the greater public.

The views are diverse according to what type of gene therapy is used and what the therapy is treating. Some people are for the use of gene therapy to treat genetic diseases and mutations. The disagreements seem to be more between the type of gene therapy used. The use of somatic gene therapy is more widely accepted than germline therapy. Most people feel that a person has the right to change their own genetic makeup, but have uncertain or undecided views on whether or not they should be able to change the future of their offspring's genetic makeup and therefore change future generations. Others, who are unsure or against the use of gene therapy bring up an important question: "Are we playing God?" Is it right to manipulate our genetic make up? Are we supposed to change the way we are created to be? God gave us the wisdom and the knowledge we need to improve and advance ourselves. The line drawn to whether or not to use this wisdom is unclear. Since we "can," does that mean we "ought" too (Bruce, 1996)? Ethical issues are always going to be in disagreement according to our individual beliefs. Other concerns of the public are the potential risks involved in the therapy itself and the harm it could cause the individuals and the possible environment effects.

Personal Opinion

I feel that gene therapy is one of the most amazing advances in the medical field. The thought of being able to correct the genetic mistakes and diseases that we have occurring in our cells is outstanding. The thought of being able to eliminate disease is very important in the survival of our existence. The thought of being able to treat diseases with the hope of cures for such diseases as Cancer, AIDS, PKU, etc. should be thought of as a "God sent." My feelings become unclear when thinking of the possibilities of using gene therapy to better ourselves in the sense of creating the "super human." I don't think that gene therapy should be used to alter our individual characteristics, for these characteristics make us different from each other and more or

less cause us no pain or harm. Even though we have been given the abilities and knowledge to perform such advances, I believe a line has to be drawn to determine how far is too far.

As for my views on somatic and germline therapy, overall I feel positive on the issue of somatic gene therapy. As for germline therapy, I feel it has the potential to be very ethically destructive. In the sense that a family has a hereditary disease that is very destructive and that disease could be prevented from affecting future generations, I feel that the use of germline therapy would be beneficial. If you could change a horrible defect or disease that is inherited generation after generation, why not fix it? As for manipulating the future of your children's physical appearance or intelligence, this seems to be over that line that needs to be drawn. I believe God created us as individuals, the genetic mistakes and diseases we are born with add to this individuality. Although I feel that God would understand us not wanting to suffer from these genetic mishaps therefore understanding our efforts to treat and hopefully cure ourselves.

In conclusion, throughout the years our technology has greatly increased in many ways, gene therapy is just one of these many amazing advances. Gene therapy has the potential to treat, create, and possibly be destructive to those who receive the therapy. Many more trials and research still needs to be done to insure its safety and effectiveness. The possibilities through gene therapy maybe endless. Decisions on how far the possibilities may stretch must be decided on and therefore be made into laws in order to protect ourselves and the future generations. The benefits of using gene therapy should not be over looked, for it has the potential to make ourselves free of disease and thus will make the difference in life and death of our loved ones and ourselves.


Biotech Applied. Gene Therapy- An Overview. Biotechnology Industry Organization, 1990. Obtained from the WWW 9/3/98: http://www.gene. com/AE/AB/IWT/Gene_Therapy_Overview.html

Bruce, Donald M.. Moral and Ethical Issues in Gene Therapy. Society, Religion and Technology Project(Church of Scotland), 1996. Obtained from the WWW 9/18/98: ww/srtproject/genthpy1.htm

Grace, Eric S.. Better Health Through Gene Therapy. The Futurist Jan- Feb 1998, v32, n1, p39(4). Obtained through Pals.

Rifkin, Jeremy. The Ultimate Therapy. Tikkun May-June 1998, v1, n3, p33(7) Obtained from WebPals on 10/6/98.

Wilson, Jim. Institute for Human Gene Therapy. Last modified: 10/20/97.Obtained form the WWW 9/3/98: /ihgt/info/whatisgt.html

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