Genetic Engineering: Gene Therapy

Table of Contents

Stem cell research is a subject that has generally been absent from the current public and political debates recently. The issue has been pushed aside by more immediate issues such as the economy, war on two fronts and healthcare.

However, it wasn’t long ago that headlines were concerned with the ethical issues of using embryonic stem cells for scientific research. Advocates as well as opponents made very verbal emotional appeals in support of their position with neither side seeming to gain much momentum.

The issue is sure to again be in the spotlight with Obama’s recent approval of limited research in this area. Much of the controversy is bound up in the use of embryos to further science. If the majority of politicians determine that this use of ‘abandoned’ embryos is ethical, the question remains where should the limit of this type of research be drawn?

Will the findings of research truly be able to combat the effects of spinal cord injuries or Parkinson’s disease? Or will it instead be akin to opening Pandora’s Box, releasing new terror on the world that can never again be contained? Are designer babies on the near horizon for the wealthy or super-humans about to be born as a result of Frankenstein-like scientific pursuits? Will science become able to make spare human parts that are superior to the original, reducing the ‘natural’ human to a level of subspecies? Where will this new technology lead? These and other unforeseen future scenarios present daunting questions that do not have clear answers and are likely not answerable.

What does seem clear is that the benefits of gene therapy to those already living seem too numerous to simply throw away in the debate. The purpose of the present study is to discover just what benefits gene therapy might have to offer present and future generations.


It is hypothesized that the current state of gene therapy technology is already capable of producing astounding results in a number of ailments and illnesses that were previously untreatable. Should this prove to be the case, it is also hypothesized that further research will prove significant in reducing or eradicating human genetic disease.


There are two broad groups of research methods available for this type of study: qualitative and quantitative. Qualitative research, according to Dawson (2007), refers to research which explores attitudes, behaviors and experiences of physical material.

It can employ evaluation of available studies, field work observations and case studies among other things. On the other hand, quantitative research refers to the use of statistical tools to conduct research, usually on a large scale. It employs hard experimental data and statistical knowledge to prove or disprove the postulated hypothesis originally set to derive the outcome of the research objectives.

Qualitative studies are focused on the examination of paradigms and perspectives of the research project. It is based on the positivism, constructivism or participatory paradigm framework. The framework is based on certain assumptions and practices which are then applied in the disciplined inquiry (Denzin and Lincoln 2005).

The complexity in the choice of research is inherent in the type of topic of research and the theory that is used for inquiry. Quantitative research methods are often adopted based on the empirical methods of structured action research with emphasis on empirical findings.

Information is defined specific to the hypothesis. On the other hand, in qualitative research, background primary and secondary research are required. It involves first hand observation as well as investigation and evaluation.

Theory employed

The process used for the current research is a combination of these methods. While hard evidence is looked to as a means of proving the relative merits of gene therapy in humanity’s attempts to eradicate genetic disease and defection, the potential of genetic research must also be viewed from a qualitative viewpoint as no hard data truly exists for future potential. As a result, this study will largely depend upon literature review to prove its argument that gene therapy contains too much potential for benefit to be ignored.


Throughout history, the benefits to society by the introduction of new medical technologies have been considerable. A primary example in the past few centuries has been the introduction of vaccines and antibiotics as a result of greater microscopic understanding of disease.

These advancements have significantly improved the well-being of people all over the globe. The science of stem cell treatments has the potential to become as significant or perhaps even more significant for human welfare than these earlier achievements in microbiology.

This field of research is on the edge of a new stage of exploration and growth that could be the forerunner of unprecedented cures and therapies in the same way that cowpox exposure was once the forerunner of today’s advanced vaccination programs.

The present enthusiasm over prospective stem cell produced remedies radiates from these new innovations in genetic biology fueled by increased understanding of the possibilities of stem cells. Though one cannot forecast exact results from basic research, there is already enough information available to suggest that a good deal of this enthusiasm is justified.

Literature review

Stem cells are basically the building block cells of a human being. They are the ‘blueprint’ cells which have the potential to develop into one of 210 different types of tissue. “Stem cells have traditionally been defined as not fully differentiated yet to be any particular type of cell or tissue” (Irving, 1999).

Although adult stem cells can be found in very small numbers within most human tissues, the majority of stem cells used for research and development can be obtained from the umbilical cord. A more precise term for these types of cells is “somatic stem cells” (Sullivan, 2004).

There are numerous potential sources for somatic stem cells. Embryonic stem cells originate from the inner cells of an early stage embryo. Embryonic germ cells can be collected from fetal tissue at a later stage of development. Adult stem cells can be obtained from mature tissues. “Even after complete maturation of an organism, cells need to be replaced.

A good example (of adult stem cells) is blood, but this is true for muscle and other connective tissue as well, and may be true for at least some nervous system cells” (Chapman et al, 1999).

Although there are numerous potential sources for these types of cells, the controversy surrounding stem cell research seems to focus on the use of unused embryos, such as those lost during miscarriage or those not used during infertility treatment, to harvest cells as they exist in the embryo in much greater quantities.

Dr. John Gearhart, professor of gynecology and obstetrics at Johns Hopkins University said of these cells, “It’s sort of like, the mother of all stem cells” (“Human Embryonic”, 1997). While the practice of organ transplant surgery from a dying patient to one who might be saved is hailed as heroic and beneficial to society, the same practice applied on the embryo level has achieved little more than criticism and contention.

There are three main objectives given for pursuing stem cell research. These include obtaining vital scientific information about embryonic development; curing incapacitating ailments such as Parkinson’s and Alzheimer’s disease and testing the effects of new drugs instead of testing on animals (Irving, 1999).

A greater understanding of embryonic development may aid in preventing birth defects and disease prior to it becoming a problem while the baby is still within the womb. It may also help settle the great abortion question in discovering just when a developing group of cells in the womb makes the transition into a human being.

Stem cell research in the area of disease prevention and treatment is also expected to aid victims of stroke, spinal cord injuries, bone diseases and diabetes (Irving, 1999). The scientific techniques for obtaining stem cells could lead to unparalleled advances and even cures for these and other ailments.

More than half of European countries and others around the world such as Japan allow for embryonic stem cell research in various degrees in recognition of the potential benefits it has to offer. Australia, for example, followed the UK’s model in allowing scientists to use the tissue of aborted fetuses, with the parents’ consent, to conduct scientific experimentation.

According to Health-Day, a daily news service reporting on consumer health, permitting stem cell research enabled Swiss physicians at the University of Lausanne to discover that a two and a half-inch piece of skin from a fetus aborted at 14 weeks could provide several million grafts that could be used to treat burn victims.

The same study also found that skin cells from an aborted fetus were able to heal burns faster than standard grafts taken from patients or adult donors.

Patrick Hohlfeld, the prime author of the study said “the use of fetal skin has tremendous potential because taking just one skin graft gives you the potential to treat thousands of people” (Strode, 2005). In light of this discovery, it seems frivolous and wasteful to allow the expiration of an unwanted embryo without also permitting the tissue to be used for more beneficial purposes.

It has been substantiated from animal research that stem cells can be differentiated into cells that will behave appropriately in their transplanted location, regardless of what that location might be. For example, the transplantation of stem cells following treatment for cancer has found much success for many years.

“Experiments such as the transplantation of fetal tissue into the brains of Parkinson’s patients indicate that the expectation that stem cell therapies could provide robust treatments for many human diseases is a reasonable one. It is only through controlled scientific research that the true promise will be understood” (Frankel, 1999).

Embryonic stem cells possess the ability to restore defective or damaged tissues which would heal or regenerate organs which have been adversely affected by a degenerative disease. Cell therapy has the very real potential to provide new cures for diabetes, cancer, kidney disease, Parkinson’s, macular degeneration, multiple sclerosis and many other kinds of diseases.

Cell therapy has also demonstrated a great potential to help repair and regenerate spinal cord injuries which would help paralyzed patients recapture lost body functions. The possibilities are limitless including greatly advancing the human lifespan because aging organs could be replenished and diseased tissue could be replaced.

Personal Opinion

Given the proven facts of gene therapy as it has been discovered in other countries and the subjective understanding of primary arguments against the use of stem cell research in this country, it seems clear that the opposition to stem cell research is based on moral fear rather than verifiable danger.

Whatever one’s moral or political position, the fact is that discarded fetuses could serve to advance scientific and medical knowledge in numerous and as yet unlimited ways. Those who believe they are taking the moral ground when it comes to the ‘unborn’ are making a choice between an already expiring group of tissues and an already living but suffering member of the world society.

Regardless of their decision, the embryo would not survive anyway while blocking research only prolongs the suffering and hopelessness of the living who might have achieved a quicker, more effective and perhaps more lasting cure as a result of stem cell research advances.

Because many embryos used for stem cell research may be acquired through the process of abortion, with laws that differ from state by state, one must take into account these definitions and restrictions as being equally applicable to the scientific community in the conducting of their research.

The vast majority of states in the union allow for abortions to be performed at least through the second trimester, which is more fully defined as 24 weeks into the pregnancy. This limitation was set based upon the neurological point of view of human development, which itself conforms to our society’s definition of death as being the absence of a cerebral EEG (electroencephalogram) pattern.

Because life is considered to be the opposite of death, this same standard is used to determine the presence of life. The presence of the EEG pattern of a fetus can be detected approximately 27 into weeks into the pregnancy. Meanwhile, an embryo is defined as a fetus that has reached approximately seven to eight weeks following fertilization.

At about four to five weeks, embryonic germ cells suitable for use in stem cell research, are developing (Morowitz & Trefil, 1992). It can be seen, therefore, that this stage is reached well before any signs of life are present at a level set far below that allowed for abortions to proceed. As an answer to the moral dilemma, however, it has been suggested that only fetuses of stillbirths be used for stem cell research.

However, the collecting of embryonic germ cells would be extremely challenging in these cases as there is only a small amount of time to collect these cells before they degenerate and become invalid. There would also be problems using these cells for research as stillbirths might have resulted from a genetic irregularity that may flaw the research (Sullivan, 2004).

While scientists continue to attempt to battle the issue of stem cell research using their wealth of data and proven human benefit, it is necessary for them to remember that they are not battling against logical, open-minded opponents but morally outraged and illogical ideologues.

Stem cell research is essential if the human race is going to take the next step forward in learning how to combat the plagues of our times – cancer, Alzheimer’s, Parkinson’s, diabetes, sickle cell. It may also provide the answer to many of our more perplexing injuries, allowing severely burned individuals to return to productive lives or those suffering from spinal cord injuries to resume normal life patterns again.

While hard facts and proven benefits are essential in determining whether this type of research should be allowed, moralists opposed to the practice need to also be reminded of what they’re standing for when they insist already dying tissue should be preserved in its natural state rather than being employed to ease pain and suffering and contributing to a better world society.


Chapman, Audrey; Frankel, Mark S.; & Garfinkel, Michele S. (November 1999). Stem Cell Research and Applications: Monitoring the Frontiers of Biomedical Research. American Association for the Advancement of Science and Institute for Civil Society.

Dawson, C. (2007). A Practical Guide to Research Methods. 3rd Ed. How to Books Ltd.

Denzin, N. K. & Lincoln, Y.S. (2005). Introduction: The discipline and practice of qualitative research.. The SAGE Handbook of Qualitative Research. Thousand Oaks, CA: Sage.

Frankel, Mark. (1999). “Stem Cell Research and Applications: Findings and Recommendations.” Stem Cell Research and Applications Scientific, Ethical and Policy Issues. American Association for the Advancement of Science and Institute for Civil Society.

“Human Embryonic Stem Cells Reported.” (July 19, 1997). New Scientist. Program on Science, Technology and Society.

Irving, Dianne N. (1999). “Stem Cell Research: Some Pros and Cons.” Written on request of Fr. Thomas King, S.J., Ph.D., Department of Theology, Georgetown University; President, University Faculty For Life, for their newsletter, UFL Pro-Vita.

Sullivan, Patricia. (2004). “Frequently Asked Questions: Do Stem Cells Come From Aborted Fetuses?” International Society for Stem Cell Research.

Strode, Tom. (2005). “Life Digest: New Stem Cell Research Encouraging but Problematic; Researchers Find New Use for Aborted Babies.” Baptist Press News.

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