Amanda Ruggles

Copyright 1996


Gene therapy is a technique which has developed in the wake of recombinant DNA (rDNA) technology. It is a process which results in the correction of a genetic disorder by the addition of a piece or fragment of DNA into the genetic material of a living, functioning cell. A mere thirty years ago this concept belonged to the realm of the human imagination made manifest in the works of science fiction. Today it belongs to the realm of the human imagination made manifest in the works of science, period. It is mind boggling to try to comprehend the far reaching effects of gene therapy. How is it affecting society? Who will benefit from its use? Should it be used at all? Should research continue? How do we answer all of these questions? The answers are not readily available, nor are they black and white, but an attempt at finding some solutions must be made. Before exploring this line of thought further, a basic understanding of the technical aspects of gene therapy is essential.

Technical Aspects

Although the highly technical aspects of human gene therapy are somewhat complex, the basic concept is very straight forward. The goal of gene therapy is to correct mistakes that have occurred within the genetic material, or DNA, of the living cell. In very simple terms, DNA is often thought of as the "language" of the biological functioning of organisms. This language is organized by letters (nucleotide pairs), words (codons), sentences (genes), and books (genomes). Before being able to repair the damaged or defective genetic material, the location of the gene or genes causing the dysfunction in the individual must be determined.

Over the last fifty years or so, scientists have made a great amount of progress in this area, including the development of techniques which allow for the controlled manipulation and replication of specific segments of the human genome. These types of techniques have come to be known as recombinant DNA (rDNA) technology and have allowed scientists to analyze functions of genes which are not necessarily directly expressed at the phenotypic level. This is done by "cutting out" or excising a particular segment of DNA of interest from the genetic material of an individual and inserting it into a bacterial plasmid (a tiny ring of DNA in addition to the normal chromosomal material found within the cells of bacteria). The excising is done with the use of restriction enzymes, which are a group of molecules capable of "cutting" the DNA at specific points along its sequence of nucleotide pairs. By observing the end products of these gene inserts, scientists are able to determine the functions of the genes themselves and are therefore better able to analyze and understand the dysfunction of certain genes at the molecular level.

Human gene therapy itself is carried out by removing cells from the tissue of the affected individual and introducing a normal copy of the defective gene or genes into these cells. This is accomplished by the use of viruses which are innately capable of transferring their own genetic material into that of their host. Viruses used for this purpose are called vectors and are of two types - disarmed retroviruses and adenoviruses. It is because of this capability of vectors to transport foreign DNA into actual cells that the prospect of gene therapy becomes possible. Once the cells are "fixed" they are reintroduced into the individual.

There are currently two types of gene therapy capable for human implementation. Germinal gene therapy consists of introducing new genetic material into the germ line cells (those cells from which the gametes are derived). This type of therapy affects not only the individual receiving the treatment but also has the ability to affect future generations through the gametes. Germinal gene therapy has not yet been executed on humans. Somatic gene therapy consists of introducing new genetic material into the cells of the body whose chromosomes will not be passed on to future generations. This type of therapy affects only the individual receiving the treatment, and has proved successful in treating human genetic disorders. The first use of somatic gene therapy on humans involved a young girl with adenosine deaminase deficiency (ADA), a rare immune -system disorder. She has been receiving treatment for this disease since the age of four, and is currently doing well at the age of nine. Since this pioneering effort, there have been many other instances of human implementation of gene therapy. In the December 14, 1995 issue of Nature, it was reported that 1024 individuals world-wide had undergone gene therapy trials.

Ethical Aspects

It is obvious that technological questions in this area are being answered very rapidly. However, the ethical questions that have arisen as a result of the technology are not being answered quite so readily. Until the development of this technology, people have had to deal with genetic inequality as a fact of life. With the advent of gene therapy, this may no longer be the case for some people. Most people feel that it is okay to use gene therapy to treat human genetic diseases. Somewhat surprisingly, even the Catholic Church has taken a stand for the use of gene therapy. Reverend Russell E. Smith, president of the Pope John XXIII Medical-Moral Research and Education Center, stated that gene therapy is "a very noble enterprise, because it is aimed at the actual cure of actual diseases." Some individuals, however, are concerned that the technique may be used for "treatment" of genetic "disorders" other than diseases. For example, in January of 1993, it was reported in USA Today that an 11 year old boy was receiving gene therapy treatments at a cost of $150,000 per year to increase his height. At 4' 11", four inches below average height, he was tired of being picked on at school for being short. His father was quoted in the article as saying, "You want to give your child that edge no matter what. I think you'd do just about anything."

Because many people are concerned about the safety of gene therapy, a special committee of the National Academy of Science was created to look into the consequences of releasing rDNA engineered organisms into the environment. The committee concluded that "there is no evidence that unique hazards exist either in the use of rDNA technique or in the transfer of genes between unrelated organisms," and that, "the risks associated with the introduction of rDNA engineered organisms are the same kind as those associated with the introduction of unmodified organisms." However, John Fagan, a professor of molecular biology at Maharishi International University in Fairfield, Iowa, is highly concerned about the fact that very little is known about the long term effects of the existence of genetically engineered organisms in the environment. To make known his concern, he returned approximately $614,000 in grant money to the National Institutes of Health. His underlying concern is that an engineering mishap with devastating effects does not occur as a result of carelessness and or lack of precaution.

Because of these and other concerns about the use of gene therapy, it has been necessary to create public policies to deal with these issues. The debate over how to regulate research and development in this area has been ongoing since the advent of rDNA technology in the early 1970's. Important issues concerning decision making and regulation of research were addressed at the now famous Asilomar Conference held in 1975. This conference, which involved mostly molecular biologists, the press, and some government officials, set the tone for dealing with rDNA public policy issues.

The major concern at the time was the possible health risks to researchers and the public at large. Many people were also concerned about the possible environmental affects. These concerns are normal and even expected with the advent of new technology; however, there existed then and to a certain extent still exists today a concern unique to the study of rDNA and its related fields. The concern is for the possible development, by some engineering mistake, of a kind of Frankenstein running rampant throughout the environment. This concern has been considered very carefully and has had a large impact on the development of public policy concerning rDNA technology.

Continued efforts in research since the Asilomar Conference have shown that the initial rDNA public health concerns no longer need be a major source of concern, as standard recombinant microbial strains have been handled safely and without incident thus far. Because of this good track record, many people now have the opinion that research involving rDNA technology no longer needs regulation, and that this regulation is in fact standing in the way of progress. Dr. Henry Miller, former head of the Biotechnology Policy Office at the FDA, has expressed his concerns about this problem and has proposed steps to rationalize the regulations of biotechnology in the United States.

There are, however, also many individuals who feel that although the current regulations may not be perfect, they are all that we have. The U.S. Department of Agriculture, which is involved in the regulation of biotechnology, responded to Dr. Miller's proposed steps by admitting that the developments in biotechnology have incredible potential for the enhancement of society, but that regulations are definitely necessary until all possible safety issues are effectively dealt with.

So how should the development and use of gene therapy be regulated, and who should be doing the regulating? I would imagine that there are as many answers to these questions as there are people on the planet. So how do we come to any decisions about anything, how do changes come about, and how do we incorporate these changes into our individual lives? I believe these things come about as a result of natural processes. For example, it is very natural for people to be interested in what controls our physical, mental, emotional and social development. It is as equally natural for people to be interested in finding cures for diseases and ailments that afflict humankind. Once these interests are carried over into research, and answers to some of these questions about our development are found, it is quite natural for people to want to regulate how these findings should be used.

It is the collective philosophy of all individuals affected by the technological developments which dictates the societal integration of those developments. I believe in this process and therefore my beliefs about how development and regulation of gene therapy should be conducted are one in the same with how these things are being conducted. I'm not saying that the issues are being dealt with perfectly or even that I agree with everything that has been done. What I am saying is that developments have been made, issues have been discussed, conferences have been held, regulations have been made, and precedents have been set. No known technology can change what has already been done.

As for the future, I cannot say what will happen. All I know is that things will continue to change, and it is my hope that in some way my personal philosophy will be incorporated into the melting pot of philosophies. By expressing my personal beliefs on gene therapy, I will be contributing to the future of this technology.

As I have said before, I believe in natural processes. Because of this I believe in continued careful and regulated research of gene therapy techniques for the benefit of individuals suffering from genetic diseases. I, like most people, do not like to see human suffering, most especially that of individuals close to me. If the technology is available to provide relief, I most definitely believe that it should be used. However, this seemingly black and white issue becomes very gray when one tries to define human pain and suffering. It is obvious to most that Parkinson's disease fit this definition, but what about the eleven year old boy who is tired of being picked on at school for being short? Should being below average height be considered a source of pain and suffering? A point must be made that practically everyone gets 'picked on' at some point in their life because of a particular physical trait. Perspective is needed here. Having to deal with being below average height simply cannot be compared to having to deal with Parkinson's disease. I do believe that it will be necessary to develop new laws and regulations to prevent industries and individuals from taking advantage of human gene therapy techniques. I understand that forming such regulations would be a very difficult and complex task, but I personally believe it to be very necessary.

There is a lot of truth to the saying that variety is the spice of life, and actually without biological variety the human population would be doomed. Therefore, it is important to be very selective and particular when deciding who should be allowed to receive gene therapy treatments. Mother Nature has done very well on her own for billions of years. We must respect her wisdom and be very prudent when contemplating making a change in her grand design.


Biotechnology Industry Organization. "Biotechnology's Impact on Society." Obtained from the WWW:

Biotechnology Industry Organization. "Speaking the Language of Recombinant DNA." Obtained from the WWW:

Blowers, Jay. "Scientist Returns Research Grant to Show Concern about Dangers of Genetically Engineered Organisms." Obtained from the WWW:

Kennedy, Dr. Donald. "Challenges to Public Policy." Obtained from the WWW:

Kevles, Daniel J. and Leroy Hood, ed. The Code of Codes. Cambridge and London: Harvard University Press, 1993.

Tanouye, Elyse. "Efforts to Repair Fetal Genes Spark Debatre aover Risks, Ethics." The Wall Street Journal 2 April 1996: B1, col 3.

U.S. Department of Agriculture. "To Regulate or Not to Regulate." Obtained from the WWW:

Wadman, Meredith. "Hyping Results 'Could Damage' Gene Therapy." Nature 14 Dec. 1995: 655.

Ward, Darrell E. "Gene Therapy: the Splice of Life." USA Today Jan. 1993: pp63-66.

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