Running  Head:  GENE  THERAPY      

 

The Risks, Costs, and Ethics of the Pursuit of Genetic Therapy in the Healing and Enhancement

of the Human Body

Kevin K. Chin

Houston Christian High School Distinguished Scholars Program

GENE THERAPY 2

 

Table of Contents

Page

Title Page…………………………………………………………………………………………. 1

Abstract…………………………………………………………………………………………... 2

Introduction………………………………………………………………………………………. 3

Review of Related Literature…………………………………………………………………….. 4

History…………………………………………………………………………………… 4

The Risks of Gene Therapy……………………………………………………………… 6

The Costs of Gene Therapy……………………………………………………………… 9

The Ethics of Gene Therapy…………………………………………………………….. 14

Conclusion……………………………………………………………………………..... 16

Method………………………………………………………………………………………....... 17

Purpose………………………………………………………………………………….. 17

Participants & Sampling Procedure…………………………………………………….. 18

Research Design…………………………………………………………………………. 19

Experimental Manipulation/Interventions/Instrumentation……………………………... 19

Data Analysis Plan………………………………………………………………………. 20

Ethical Consideration……………………………………………………………………. 21

Bias……………………………………………………………………………………….21

Assumptions……………………………………………………………………………... 21

Limitations………………………………………………………………………………. 22

Findings…………………………………………………………………………………………. 22

Sample…………………………………………………………………………………… 22

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Survey Findings…………………………………………………………………………. 24

Analysis…………………………………………………………………………………. 27

Discussion………………………………………………………………………………………. 28

Conclusion……………………………………………………………………………………… 32

Remaining Questions…………………………………………………………………………… 33

Nutritional Supplements………………………………………………………………… 34

Genetic Pedigrees………………………………………………………………………... 35

Biblical Connection………………………………………………………………………………37

References………………………………………………………………………………………. 39

Appendix………………………………………………………………………………………… 42

       

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Abstract

The researcher examined gene therapy and its risks, costs, and ethics. While this review of related literature focuses on what relevant scholarly sources have to say about the downsides of genetic therapy, the researcher does not ignore gene therapy’s proclivity toward success in the future. After researching the three aspects of gene therapy, the researcher conducted new research centering on the majority’s opinion toward cost and ethics, overlooking risk because of its natural decline with further research. Thus, two null hypotheses were composed: the first involved cost and the second, ethics (where gene therapy was divided into its two components). The researcher gathered data through a 10-question survey sent to a convenient sample population and ultimately concluded (1) gene therapy was not cost-effective, (2) gene healing was ethical, and (3) gene enhancement was not ethical. Researching gene therapy directed the researcher’s attention towards the pursuit of perfection through nutritional supplements and genetic pedigrees and how these two industries have impacted the United States economically and socially. The nutritional supplement industry has positioned itself for success in the United States’ economy, reaching sales up to $11.5 billion as of 2012 and providing 450,000 jobs. Genetic pedigrees have been on the rise as doctors and patients now acknowledge the benefits of having them as “screening tools” for potential diseases. By looking into these two industries, the researcher communicates the idea gene therapy can also influence the world positively.

GENE THERAPY 5

 

The Risks, Costs, and Ethics of the Pursuit of Genetic Therapy in the Healing and Enhancement

of the Human Body

As the human race continues to suffer from the endless appearances of new mutated

genes, scientists have turned their focus on a new and promising “cure”: gene therapy (“Anti-

cancer…,” 2009; Cotrim & Baum, 2008). Nowadays, scientists know that a missing or an extra

gene can cause diseases, also known as mutations (Cotrim & Baum, 2008; “...Genetic

Medicine,” 2009). Destroying the function of a gene, the mutation will produce defective

results, which includes malfunctioned proteins and uncontrolled growth (Cotrim & Baum, 2008;

“...Genetic Medicine,” 2009). On the other hand, gene therapy replaces and corrects mutated

genes, achieved through the “insertion of a functioning gene into cells to correct a cellular

dysfunction or to provide a new cellular function” (Cotrim & Baum, 2008). Moreover, the idea

of gene therapy has actually existed for over a century, but after the recent breakthrough in

recombinant DNA technology gene therapy has became a reality (Cotrim & Baum, 2008). As a

result, gene therapy carries the potential to be the key to permanent victory over the lifelong

battles with the malicious diseases that have plagued the human race for centuries. While current

medicines––surgery, drugs, chemotherapy, and other methods––are temporary or ineffective

solutions in the fight against present diseases, scientists bring the battle to the level of genetics.

As a result, the treatments become more effective but also more dangerous. The researcher

considers the dangers of gene therapy by asking, is gene therapy a worthy pursuit when the

researcher considers the risks, the costs, and the ethics of gene therapy?

GENE THERAPY 6

 

History

The studying of genetics officially began in the 1800s with the Austrian monk Gregor

Mendel, theorizing that a relationship between genes and traits existed (“...Genetic Medicine,”

2009). As he conducted his own research in a monastery garden, Mendel observed how traits are

passed from one generation to the next (“...Genetic Medicine,” 2009). Mendel, dying in 1884, set

the foundation for the study of genetics (“...Genetic Medicine,” 2009). In 1906, zoologist

William Bateson coined the term––genetics, which is the study of heredity (Wynbrandt &

Ludman). Nevertheless, even before Gregor Mendel, many early individuals had observed

genetics. The Egyptians, the Babylonians, the Chaldeans, and the Ashkenazi Jews noticed

similarities between blood-related individuals and unique differences between unrelated

individuals (Wynbrandt & Ludman). The Ashkenazi Jews understood and acknowledged that

the third child would also have hemophilia after the first two infants died of the same disease

(Wynbrandt & Ludman). Ultimately, the Jews successfully connected the defects in genetics to

diseases.

Nonetheless, modern genetics officially began with James Watson and Francis Crick’s

discovery of the structure of deoxyribonucleic acid, or DNA, in 1953 (Editors of Salem Press,

2008; “...Genetic Medicine,” 2009). Since then, scientists have sought a way to correct genetic

defects in a cell, ushering in the idea of gene therapy (Editors of Salem Press, 2008). By the

early 1970s, scientists could isolate and experiment on specific strands of DNA by the use of

“restriction enzymes,” which cut DNA molecules at specific points (Wynbrandt & Ludman). In

doing so, the isolation of the strands assisted the search for specific genes (Wynbrandt &

Ludman). Scientists then identified the specific functions of certain genes and marked the

location of the genes on the chromosomes (Wynbrandt & Ludman). In the 1980s, scientists

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replaced defective enzymes in the metabolic pathways (Editors of Salem Press, 2008). One

successful experiment includes treating babies (but can be found in adults as well) with

Adenosine deaminase (ADA) deficiency–a disease where an enzyme no longer converts the

toxins into uric acid causing immunodeficiency (Editors of Salem Press, 2008). Stem cells were

removed from the umbilical cord of a child with ADA deficiency, and correct genes are inserted

into the collected stem cells; finally, the altered cells are inserted back into babies (Editors of

Salem Press, 2008). After many years of examining the babies, the scientists established gene

therapy as a cure for ADA deficiency (Editors of Salem Press, 2008). Nowadays, scientists are

trying to use gene therapy in the fight against multiple diseases, such as cancer,

neurodegenerative diseases, and hemophilia.

Though gene therapy holds much promise, it still has its own setbacks. Insertion, which is

the process of “inserting” the new gene, uses two types of vectors: viral, also known as

transduction, and nonviral vectors, also known as transfection (“Anti-cancer...,” 2009; Cotrim &

Baum, 2008). Though viral has a better success rate of introducing the correct genes into the

patient’s DNA, viral vectors pose many safety concerns like the possibility of prompting an

immune response; on the other hand, nonviral vectors are inefficient (“Anti-cancer...,” 2009;

Cotrim & Baum, 2008). Gene therapy has been a slow process filled with its own tragedies, such

as the death of eighteen-year-old Jesse Gelsinger in September 1999 (Cotrim & Baum, 2008).

Another specific problem with gene therapy deals with cancer, a disease that has been observed

in the human race for at least a hundred years (Cross & Burmester, 2006). Similar to other gene

therapy processes, cancer treatment, using gene therapy, includes the introduction of engineered

cells–a recombination of dead cancer cells and cytokine genes or antigenic protein genes–to the

patients, allowing their immune systems to recognize cancer cells (“Anti-cancer...,” 2009; Cross

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& Burmester, 2006). With gene therapy, the fight against cancer seems to be a clear victory;

however, initial trials “have produced mixed results”—condition improved, condition worsened,

or no effect—showing how gene therapy still needs time to develop (Cross & Burmester, 2006).

Like many other medical practices, the purpose of gene therapy is to return the human

body to the state of “perfection.” In fact, gene therapy has the potential to enhance human traits

or qualities by adding or correcting genes. Therefore, this ability brings many people to wonder

whether or not gene therapy is actually ethical (Cotrim & Baum, 2008). While gene therapy is

only in its early stages, it has already encountered major disappointments. In the end, this

researcher must consider risks, costs, and ethics in order to present a sufficient argument for or

against gene therapy.

The Risks of Gene Therapy

Rate of Failure

From the beginning, scientists recognized that bringing the battle against diseases to the

level of genetics required an enormous amount of caution. Since inserting genes carries the

possibility of activating other genes that could cause diseases, scientists constantly worry about

the possibility of inserting a gene in or near healthy genes (Cohen, 2003; Coghlan, 2010). In

other words, gene disruption became a major concern among gene therapists. Four French boys

fell victim to this when they were treated for immune deficiency and developed leukemia

(Coghlan, 2010). As a result, one of the boys died. From this experiment, Mark Kay of Stanford

University stated, “[Gene disruption] raises the bar of risk somewhat. But how much higher it is

raised is still unclear” (Cohen, 2003).

Mark Kay continues to research on mice with a new vector, adeno-associated viruses–

another popular vector among gene therapists (Cohen, 2003). Unfortunately, his conclusions

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were quite discouraging to the gene therapy activists. Injected in mice livers, the adeno-

associated viruses integrated themselves near active genes, which are working genes (Cohen,

2003). In fact, out of 29 insertions, 72% appeared in the genes (whether healthy or mutated)

rather than junk, non-functioning DNA (Cohen, 2003). Furthermore, 20 of the genes were active

genes, or healthy genes (Cohen, 2003). In the end, Kay was forced to conclude that active genes

are “natural hot spots” for inserted genes, and therefore, gene therapy may hold higher risks than

thought (Cohen, 2003). If the inserted genes do disrupt active genes, then gene therapy may do

more harm than good.

The harm can be clearly seen in the deaths of patients. Jesse Gelsinger, who died in

1999, was not the only victim of gene therapy (Skelly, 2007). Treated in a rheumatoid arthritis

trial, a 36-year-old Jolee Mohr also fell victim of gene therapy’s tendency to fail (Skelly, 2007).

On October 2005, Moher and 126 other subjects received an initial dose of a placebo (a pill), and

then 74 of them received a second dose of another drug called tgAAC94 (Skelly, 2007). On the

second dosage, Mohr died in the University of Chicago Medical Center (Skelly, 2007). As a

result, U.S. Food and Drug Administration put gene therapy on hold to research the dangers of

adeno-associated viruses (Skelly, 2007). In essence, scientists such as Mark Kay can conclude

that adeno-associated viruses are the cause of Jolee Mohr’s death, developing histoplasmosis––

fungus infection––by suppressing the tumour necrosis factor alpha receptor (Skelly, 2007). Yet,

doctors and researchers continue to support the use of adeno-associated viruses, focusing on the

great potential gene therapy carries. For instance, Dr. Chris Evans is one of many who plan to

conduct his own research with adeno-associated viruses (Skelly, 2007). While Jesse Gelsinger

most likely died from an immune reaction to the adenovirus, Jolee Mohr most likely died from

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developing a fungus infection (Skelly, 2007). As a result, gene therapy was put on hold for a

short time.

Despite the risks that come with adenovirus vectors, scientists continue to be proponents

for gene therapy (Wenner, 2008). Instead of contemplating the potential dangers of gene therapy,

scientists optimistically and persistently explore and experiment to understand the “full

dimension of what [gene therapy] can do” (Wenner, 2008). In the United States, gene therapy

still has not obtained approval from the Food and Drug Administration; nevertheless, it has 800

trials ongoing (Wenner, 2008). So far, the efforts in curing diseases seem to be paying off.

Scientists have just begun to realize the potential benefits of gene therapy through countless

breakthroughs in curing cystic fibrosis, restoring sight to the blind, attacking cancer cells, and

repairing the heart (Wenner, 2008).

Delivery Issues

Despite these breakthroughs, scientists have also begun to acknowledge the greatest

barrier in gene therapy–the delivery (Wenner, 2008). Unlike other medicinal processes that have

only a small amount of the pill or the injection successfully reaching the target, gene therapy

targets only the desired tissues (Wenner, 2008). Thus, after the recombinant gene is inserted into

the body, the whole gene must be delivered to the right location to be able to target the correct

tissues (Wenner, 2008). To make things more complicated, the desired locations may be totally

inaccessible targets (Wenner, 2008). Therefore, the delivery to the right location is the first

major problem in gene therapy. Secondly, the therapeutic contents are stored within modified

viruses–the vectors of gene therapy–that may cause the immune system to react, to fight, and

ultimately, to reject the delivery (Wenner, 2008). Scientists counter the immune system’s efforts

by using lower therapeutic doses or by pretreating patients with immunosuppressive drugs or by

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essentially hiding the vectors (Wenner, 2008). Other scientists have tried to use vectors with

“naked” DNA, unrecognizable to the immune system (Wenner, 2008). Even though gene therapy

contains these two difficult hurdles, scientists continue to remain optimistic and focused on the

overwhelming potential of gene therapy, searching for answers to overcome these hurdles once

and for all.

In the end, scientists have entered the world of genetics with caution. Many have come to

realize the many problems preventing gene therapy from being successful. Among these

problems are gene disruption, which can lead to fatal consequences; the delivery to the correct

location; and the immune system’s rejection of vectors carrying the therapeutic genes.

Furthermore, the deaths of patients have also tainted and slowed the process. Still, scientists

remain optimistic, stubbornly searching the unknown of possibilities that come with gene

therapy.

The Costs of Gene Therapy

Actual Treatments

After presenting the possibilities and the problems of gene therapy, a common

controversy resides in the major issue of price: is gene therapy too expensive for the common

man? Though limited amount of quantitative information can be found on the Internet, this

researcher presented today’s prices to the best of his abilities, the benefits of gene therapy, and

the idea of intellectual property. After acknowledging the price and other related information,

this researcher reached the conclusion that gene therapy would, in fact, be improbable to attain

for a common patient in today’s society.

The Glybera treatment treats for lipoprotein lipase deficiency (LPLD)–the inability to

break down fat (Staff Writer, 2012; Whalen, 2012). As a result, abominable pain and pancreatic

GENE THERAPY 12

 

inflammation might occur (Staff Writer, 2012; Whalen, 2012). To evade these agonies, a virus

with a functioning copy of the gene for breaking down fats infects the individual’s muscles (Staff

Writer, 2012; Whalen, 2012). This process costs $1.6 million per patient; thus, this therapy is

cost-prohibitive to anyone except the wealthy (Staff Writer, 2012; Whalen, 2012). More

importantly, this treatment became the first gene therapy drug that the European Union has

approved–a huge milestone in medicine (Whalen, 2012). This accomplishment did not happen

without setbacks, including three rejections from the European Medicines Agency (Whalen,

2012). Dutch Company UniQuire, a major contributor to the development of this drug, claims

that this one-time injection of the drug is worth the $1.6 million because of the small market: one

or two people per each million suffer from LPDP (Whalen, 2012). Furthermore, national

insurers in Europe are likely to pay the bill (Whalen, 2012). With that said, the price is still

being criticized by many, just like the prices for drugs of other rare diseases. In the end, this

price may still be out of reach for those with LPDP (Whalen, 2012). Despite the arguments with

the price, UniQuire plans to obtain approval in the United States, where no gene therapies have

been approved for the market, in 2013 (Whalen, 2012). Glybera marks only the beginning of the

era of expensive gene therapy drugs.

Another example is gene therapy for a rare, genetic brain disorder that causes severe

astrophy (Lopatto, 2012). While a normal baby born with a brain disease would have severe

deterioration and death within about three years, studies have shown that gene therapy holds the

ability to increase each baby’s life from seven to 10 years (Lopatto, 2012). Approved in 2001,

this study would transport the adenovirus with the healthy genes through a hole drilled inside the

skull (Lopatto, 2012). The results showed that this therapy was more successful in younger

patients than in older patients (Lopatto, 2012). With an improved alertness, the younger patients

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still retained “cognitive abnormities,” like the inability to talk at all (Lopatto, 2012). An older

patient improved from a vegetative state to a responsive state with blinking as “yes” and opening

her mouth as “no” (Lopatto, 2012). In the end, 11 out of the 13 patients had a decrease number of

seizures; the other two patients have had none before and after the procedure (Lopatto, 2012).

As a result, the treatment does extend life and restores some functions in the brain, but the

procedure costs $100,000 just for the first two years.

Why do such treatments cost so much? This question touches the beneficial aspects of

genetic therapy. Since gene therapy is commonly thought to be permanent, the cost could be

justified (Staff Writer, 2012). In a sense, gene therapy has the ability to cure all kinds of cancer,

autism, and other diseases, ultimately prolonging life in the future (“Do the benefits

outweigh…,” 2013). This capability poses many questions concerning when and how death

would occur and the concept of the “survival of the richest.” Additionally, gene therapy aims to

correct patients’ childhood deformities, which eventually leads to the beautification of any

person with enough money to pay for a specific desired trait (“Do the benefits outweigh…,”

2013). The issue of price remains a personal opinion that can be swayed depending on an

individual’s income and opinions on the pursuit of longer life and beauty.

Intellectual Property

Another issue concerns intellectual property. As of right now, only the major powers of

the world––the economically successful––hold the ability to research gene therapy, while the

poor countries have the facilities and the expertise but not the money (Bouguerra, 2005). For

example, India owns six laboratories but remains dependent on rich countries for the funding

(Bouguerra, 2005). In essence, the major powers fund the poorer countries if they are willing to

research according to the major powers’ agenda. Therefore, Indian scientists are forced to

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identify human genes instead of organisms that will benefit their own population–microbes,

mosquitoes, contaminants, and other organisms (Bouguerra, 2005). By researching their desired

organisms, they can develop applications for the poorer societies, while DNA research with

humans would not necessarily lead to medical treatments for the poor (Bouguerra, 2005). In

essence, the rich can dictate what is being researched and continually obtain intellectual property

from the poor’s cheap labor. This dilemma leads the researcher to ask an important question:

would gene therapy be available or even beneficial to the poor population in rich nations? “No”

would probably be the answer because gene therapy is unaffordable and unavailable to the poor

(Bouguerra, 2005; Gert, 2001). In a way, the priority lies in caring for the rich rather than the

poor.

Genetic Modified Foods

Since gene therapy is basically the genetic engineering of humans, this researcher can

study the history of genetically modified foods to predict the future of gene therapy. In fact, the

two processes share similar characteristics, allowing this researcher to conclude many of the

problems with gene therapy were problems for genetically modified food. In genetically altering

a plant, desired genes are also inserted into the plant’s chromosomes (Teresko, 1997). In fact,

one purpose of genetically altering a plant’s genes is to give the plant the ability to produce its

own insecticides (Strom, 2013). This purpose can parallel with gene therapy’s potential of

fighting malicious cancer cells; however, people remain in a heated debate on the ethics of gene

therapy. On the other hand, genetically modified organisms (GMOs) have crept into our society

in massive amounts without public awareness (Black, 2012; Strom, 2013). The truth is 80% of

processed foods in the grocery stores are GMOs (Black, 2012). In conclusion, the widespread

use of implementing GMOs foreshadows the widespread use of gene therapy in the future.

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Furthermore, this researcher can compare in terms of price. In an article from alive.com,

the author reveals how even the farmers of GMOs are not earning a profit (Abergel). Engineered

corn has cost farmers $659 million, while the profits only amounted to $567 million (Abergel).

Thus, the farmers are actually losing money. Where is the extra money going? Biotech

companies. These research companies continue to earn a substantial amount, which means the

farmers and consumers are still a big portion of the expenses for GMOs (Abergel). In

comparison to gene therapy, this researcher can suspect gene therapy would continue to be an

expensive market, while the genetic research companies receive the most benefits from gene

therapy’s success.

When put into perspective, genetic therapy treatment demands a substantial amount of

money from patients. Consequently, only the wealthy class of Americans would have the

resources to spend in order to reap the benefits of gene therapy. Furthermore, with the issue of

intellectual property, the researcher can see how rich communities fund current research and

development; therefore, the benefits are aimed toward only the rich communities. Even when

changing a physical trait or curing a disease becomes necessary, would the lower class be able to

afford it? The answer is probably not. Also, while GMOs in foods were unwelcomed at first,

they are now common and consumed by many people across the globe. On the other hand, the

price remains an issue for both GMOs and gene therapy, but the consumers still purchase GMOs

despite the cost. This comparison only suggests gene therapy, despite its potential dangers and

unethical possibilities, would gradually be accepted in the future.

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The Ethics of Gene Therapy

Exploitation of Wealth

While gene therapy’s main purpose is to cure the world of malicious mutations, gene

therapy’s potential goes far beyond. In today’s world, wealthy individuals tend to pursue vain

medical treatments, such as plastic surgeries. This aspect brings a graver danger in gene therapy–

the possibility to insert any desired genes in order to add or to correct any characteristics of any

human being (Anderson, 1999). Humans would be able to abuse the potential of gene therapy to

the point where “eugenics could be practiced on a scale far larger than any ‘selective breeding’

policy could accomplish” (Anderson, 1999). In other words, humans (who can afford the

treatment) would all become “perfect” in a matter of a few procedures. Wealthy individuals

would always have the irresistible temptation to reach the shifting standards of beauty in society

(Anderson, 1999).

These wealthy individuals will be the future targets for gene therapy. In 10 more years,

global life science companies will hold patents on about 30,000 genes (Rifkin, 2006). Even

today, genes are the “raw resource[s] of the new economic epoch” for a variety of businesses

dealing with agriculture, animal husbandry, and pharmaceuticals (Rifkin, 2006). With the

installment of gene therapy, human medicine will intensify the commercial use of genes. A

market driven by eugenics––the science of improving a human population––would become

enormously plausible (Rifkin, 2006). Even today, indications of potential abuse are appearing

(Anderson, 1999). For example, one company intended to develop a treatment for hair loss for

those who suffer from chemotherapy (Anderson, 1999). Finding a gene that could stimulate hair

growth to prevent traumatic effects of chemotherapy’s hair loss seems to be a genuine motive;

however, after some investigation, the real motivation lies in the ability to market this gene to

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naturally bald men (Anderson, 1999). Is this a bad alternative motive? Not really, but where is

the line dividing unethical from ethical? Instead of gene therapy, the abuse can lead to genetic

enhancement (Gert, 2001). Therefore, it is essential to remain cautious as scientists continue to

make breakthroughs in gene therapy.

Gene Enhancement

Another problem to foresee is the parents’ ability to genetically alter their babies before

birth to obtain traits for strength, intelligence, resistance to toxins, and many more (Gert, 2001).

Conversely, scientists do realize the potential danger of genetic enhancement. By enhancing one

generation’s genetics, the effects do not last only one generation; the enhanced genes would be

passed on to future generations, and thus making the effects endure for many generations to

come (Gert, 2001). Therefore, a slight mistake in gene enhancement will be detrimental to the

future society (Gert, 2001).

Since gene enhancement would be an expensive luxury, another concern stems from gene

therapy playing a role in inequality and abuse (Gert, 2001). Wealthy families would be able to

purchase their “social and political dominance” over others (Gert, 2001). Throughout history,

certain traits would give an individual a certain edge in life. For example, if gene therapy existed

in the age of Hitler, the persecuted Jewish population might consider giving themselves blue

eyes and blonde hair. Then, they could avoid persecution and could enter the ranks of the Aryan

population. Now, the question is whether or not the individuals will take the risk in bankruptcy

or other sacrifices to obtain gene enhancement–the gateway to a better life. The consequences of

such a future would be incomprehensibly harmful. The possibilities do not stop there. Gene

enhancement can engineer humans for special “niches” in societies (Gert, 2001). For example,

super soldiers, like Captain America, from science fiction could become a reality. How about

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engineering poison resistant immune systems, see-in-the-dark eyes, perfect bodies suited for

runners? The possibilities are limitless; the consequences are unfathomable. Nonetheless,

scientists might not to have these risks in mind as they compete to be the first to discover the

new possibilities of gene therapy (Gert, 2001).

With gene therapy, gene enhancement becomes the next step. While many gene therapy

scientists and advocates ensure the public that genetic engineering would be limited to genetic

repair, the temptation to improve oneself is too much to bear for certain individuals (Gert, 2001).

In other words, if scientists continue their pursuits in gene therapy, genetic enhancement is

inevitable. Some scientists believe the only way to postpone the inevitable is to maintain focus

of “repairing” genes rather than “replacing” genes (Gert, 2001). Yet, what if a “damaged” gene

was being short instead of being tall? In a world with gene therapy, humans can allow their

imaginations to go wild, picturing a future where 73-year-olds and 82-year-olds would still be

considered youths (Anderson, 1999). While researchers have this picture in their minds, they

might as well also have the picture of extreme inequality and segregation and mistreatment of the

lower class. The future is literally in the hands of humans (mainly the rich). Whatever

experiment conducted in gene therapy would then be a step closer to gene enhancement. In

today’s society, there remains a crucial and fundamental question: Will man in the present and

the future remain genetically responsible? Hopefully, the answer is a resounding “yes.”

Conclusion

Modern genetics began in 1953 with the discovery of the double helix. From that stage,

scientists have identified genes, part of the Human Genome Project. Presently, genes have

become the focus for a cure for many diseases through a process known as gene therapy. In

layman’s terms, gene therapy is the insertion or altering of genes in humans. Gene therapy holds

GENE THERAPY 19

 

many possibilities, including curing practically any disease or mutation and changing human

traits. Nevertheless, gene therapy is still in its trial stage and remains expensive. Furthermore, the

chance for failure remains a high concern among researchers. Mainly, the problem lies in the

delivery of the gene. Would it be accepted? Would it end up in active or junk DNA? After

perfecting the tendency to fail, the next obstacle becomes price. Today, a gene therapy treatment

like Gybera costs around a million dollars. This observation brings the question “who would be

gene therapy’s main consumers?” The rich. In essence, gene therapy would only contribute to the

“survival of the richest.” The upper class would be able to cure their diseases and change their

appearances, which brings to question whether or not pursuing a long life (to the point of

immortality) and beauty (in terms of perfection) is ethical. In the end, if researchers continue to

pursue after gene therapy, man would be able to control many aspects of God’s creation. God,

however, warns us not to be obsessed with our physical appearances but rather our true spiritual

condition. Most likely, the beautification of our physical appearance will not glorify God, but

prolonging our life can lengthen our time to serve the Lord on this earth. Nevertheless, in all

things, man must submit his will under God’s supreme authority. Therefore, if gene therapy

becomes a major part of the future, man must be ready to take on the responsibilities that come

with genetic enhancement and genetically extending life.

Method

Purpose

After looking over all the risks, costs, and ethics of genetic therapy, the researcher

decided to focus on the costs and ethics aspects of gene therapy. The reason for the exclusion of

risk as a research focus was because the number of risks in gene therapy would most likely

decrease in the future, as further testing and research would continue to perfect the process of

GENE THERAPY 20

 

inserting genes or correcting genes. Next, this researcher hoped to understand the majority’s

opinions on the current cost of gene therapy treatment (Glybera at a cost of $1.6 million) and the

ethics of gene therapy. As a result, the researcher tested two null hypotheses.

First Null Hypothesis

In the majority’s opinion, gene therapy is cost-effective.

Second Null Hypothesis

In the majority’s opinion, gene therapy is ethical.

In other words, the researcher studied the people’s views on gene therapy’s cost and ethicalness

to see in what circumstances would the majority most likely accept gene therapy today.

Participants & Sampling Procedures

The researcher used a convenience sampling method because of the limited number of

people to whom the researcher could send the survey. As a result, this sampling method results

in many problems when making generalizations. Thus, the researcher included questions on the

population’s age, ethnicity, occupation, personal monthly income, estimated amount spent on

medical treatments, and religion to better understand the population tested. These questions

allowed the researcher to qualify the generalizations and conclusions made while presenting the

data collected.

With that said, the ideal sample population would consist of a diverse expanse of ages,

ethnicities, occupations, and religions, mirroring a sample population from a random sampling

method. The main two characteristics that the researcher hoped to obtain a wide variety of

would be occupation and religions. Occupation diversity would guarantee a sample population

with different personal incomes; thus, when the survey participants answer the question over

price, the results to that question would not be biased. Similarly, since religious backgrounds

GENE THERAPY 21

 

often contribute to a person’s morality, religious diversity would guarantee a sample population

with different ethical views. Therefore, an ideal sample population would be that of different

occupations and diverse views of ethics.

Research Design

Using a survey made through Google Forms, the researcher conducted a descriptive study

through a quantitative research design. In other words, the survey produced data that was

analyzed through a statistical investigation of patterns and trends in the situation of whether gene

therapy is cost-effective and ethical. The advantageous reasons for picking this research design

include the ability to reach a large population and the ease in collecting data. As the world today

is now electronically connected through the services of social media sites and email, the

distribution and also collection of data becomes exponentially easier and faster.

Experiment Manipulation/Interventions/Instrumentation

As said before, the researcher designed the instrument used for collecting data––a survey

or questionnaire––containing mostly multiple-choice questions. The researcher used a survey for

its efficiency in obtaining responses at a rapid rate. Thus, with more responses, better estimates

of the population’s true opinions were made. In terms of the cost-effectiveness null hypothesis,

the researcher included two questions; for the ethics null hypothesis, the researcher included two

questions as well. The first question dealing with cost-effectiveness provided the same

information (the price of Glybera) to all survey participants, hoping to receive their “yes” or “no”

answer of whether or not they would pay for such a treatment. The second question for cost-

effectiveness asks the participants to use the cost of their past medical experiments to produce an

ideal price range for gene therapy. This question does however include a flaw, since the need for

major surgeries for injuries, the rise of severe illnesses like cancer, and other circumstantial

GENE THERAPY 22

 

situations would cause an inflation on the annual amount spent on medical treatments.

Nevertheless, the ability to control such a flaw is improbable. Since gene therapy could be

divided into two parts––healing and enhancement––the questions dealing with ethics offered the

definitions of gene healing and gene enhancement (formed by the researcher from the research

on current literature on gene therapy) to all the participants. As said before, the researcher

expected to use the variety of beliefs held by specific religions and morals developed through

certain cultures to develop conclusions that generalizes the public’s opinion.

Data Collection

This survey or questionnaire was distributed by posting the link to the survey on the

social media site Facebook and through emailing the link to Houston Christian High School’s

parents and faculty along with family and friends of this researcher. Also, this researcher

expected individuals who completed the survey to also share this link. The time this survey was

available to the public began on October 21, 2013, and ended December 3, 2013. No reminders

were given in the process of collecting responses to the survey.

Data Analysis Plan

The researcher conducted analysis on the collected data from the survey through

inferential statistics. Utilizing a one-proportion z-test for each null hypothesis, where p equals

the proportion of the population who believes gene therapy is (1) cost-effective or (2) ethical. If

p was significantly greater than or equal to 50%, then the researcher would not be able to nullify

the null hypothesis tested. If p was significantly less than 50%, then the researcher had compiled

enough evidence against the null hypothesis to validate the alternative hypothesis.

GENE THERAPY 23

 

Ethical Consideration

In order to protect the human subjects, the phrase “taking this survey indicates your

consent for your response to be used in my research” was included in the email and also the

survey sent to the public. Since the population’s views may change in the future and some views

may seem offensive to others, the researcher will not hold the responses against the human

subjects. Furthermore, the responses remained anonymous to prevent emotional or physical

harm to the subject when the conclusion of the data collected is presented.

Bias

Since the link to the survey was distributed through a social media site and multiple

emails, the study design and instrument presented a bias against people without access to

computers. Furthermore, the survey was in English, creating a bias against people who are

illiterate in the English language or even blind. Also, since a majority of the responses came

from an email to Houston Christian High School’s faculty and staff, the data collection process

naturally created biases. Furthermore, access to the link on Facebook was limited to only the

researcher’s “friends.” These “friends” most likely held similar views and were raised in similar

circumstances as the researcher. As a result, the study design was bias towards Christians, the

richer population, and the Caucasian population.

Assumptions

In order to create a substantial generalization of the public’s responses, the researcher

held many assumptions to test the null hypotheses. For example, the researcher assumed that the

participants would be honest in their responses. Furthermore, the assumptions of a typical

population and normal distributions also were made in the process of analyzing the data

collected.

GENE THERAPY 24

 

Limitations

Because of limitations on time, money, and resources, the researcher deliberately did not

study the public’s opinions on the risks of gene therapy as these opinions are subject to change as

the risks are likely to decrease in the future. The continual trials for gene therapy would only

further the perfection of the process. Also, since there are many different gene therapy

treatments––each with their own risks––the amount of information to explain to the general

public would be overly tedious as well. The researcher also avoided the topic of gene therapy’s

cost in the future as that is subject to change as well. If gene therapy treatments came in the size

of pills, then the supply would be greater, creating a lower cost. Thus, in conclusion, there are

simply too many uncertainties regarding the future in general, so these aspects of the problem

remained outside the researcher’s interests. On the other hand, the researcher could have

improved the research design and sampling size by having access to the costs of current trials for

gene therapy and a larger, more diversified population.

Findings

Sample

In total, the researcher obtained 196 responses. In terms of the sample reached in this

researcher’s experiment, the age ranged from 15 to 65, with the understanding that some did not

answer the age question and also the researcher had to discredit one improbable answer of

300,000. For ethnicity, the survey tended to reach mainly Caucasians (72% of the population)

with Asian or Pacific Islanders running in second (12% of the population).

GENE THERAPY 25

 

Occupation ranged from all sorts of fields (shown in Figure 1A), showing the survey’s

success in obtained answers from a population with a widespread of fields. The “other” category

would include “occupations” such as homemakers or students.

Figure 1A

Next, a plurality of the sample earned more than $10,000 a month (46% of the

population) and was predominantly Christian (93% of the population) as shown in Figure 1B.

Figure 1B

Trades  and    Personal  Services  

Science,  Math,  &  Technology  

Public  &  Social  Services    

Media  &  Social  Services  Health  &  Medicine  

Other  

Arts,  Entertainment,    &  Sports    

Business    

GENE THERAPY 26

 

The “other” category included a Jehovah Witness and several Catholics. Lastly, the population

spends a wide range for medical treatments, with the majority spending in the range of $1,000 to

$4,999 per year on average in the last three years. Knowing all this background information of

the sample greatly helps the development of an accurate scope to the conclusion.

Survey Findings/Interviews/Observations

After the questions over the sample’s background information, the next two questions on

the survey focused on the first null hypothesis over price. As stated previously, since gene

therapy was only in its trial stages and not many treatments have reached the market, information

on gene therapy’s price was hard to obtain. Nevertheless, a gene therapy treatment known as

Glybera recently reached the European markets at a price of $1.6 million dollars. Thus, the

researcher utilized this information and formed the question, “Would you pay the price of $1.6

million dollars if a close family member had LPLD?” In addition to this question, the researcher

desired to put this price into context by explaining what was LPLD, the reasons for its high price,

and current ways of treating this rare disease (which is basically to eat a low-fat diet).

Figure 2A

As seen in Figure 2A, 89% of the sample replied “no” to this question; therefore, the researcher

could conclude that the majority of the sample would not pay for the LPLD treatment and would

prefer enduring a low-fat diet instead.

GENE THERAPY 27

 

As the majority would not pay for a $1.6 million dollar treatment, the researcher desired

to obtain the public’s ideal price for gene therapy by having the participants answer the

following question “What would be your ideal price range for genetic therapy treatments?” after

comparing with the cost of past medical procedures. The responses to this question varied from

all ranges set by the researcher, which can be seen in Figure 2B.

Figure 2B

Most importantly, the researcher noticed the price range from $1,000 to $9,999 held 37% of the

vote, more than any other price range.

The last two questions revolved around the second null hypothesis over ethics. In

summary, gene therapy treatments can be divided into two classifications: healing and

enhancement. Consequently, in order to determine gene therapy’s ethicalness, the researcher

decided to acquire the survey participants’ opinions on whether each classification was ethical or

unethical. As a result, this inquiry produced the two questions (1) “Is genetically healing

ethical?” and (2) “Is gene enhancement ethical?” With these questions, the researcher also

provided definitions. Gene healing was “the changing or replacing of a faulty gene to heal a

disease,” while gene enhancement was “the adding or changing traits to the human body.”

GENE THERAPY 28

 

Figure 2C

According to the results found in Figures 2C, an overwhelming 91% of the sample believed gene

healing was, in fact, ethical.

Figure 2D

According to the results found in Figures 2D, 35% of the sample believed gene enhancement was

ethical. So, as long as gene therapy never crosses the line from healing to enhancement, the

majority of the sample would support gene therapy and its many benefits.

GENE THERAPY 29

 

Analysis

In order to statistically analyze the responses from the survey, the researcher performed a

one-proportion z-test. If the p-value obtained was less than .05, the researcher would reject the

null hypothesis.

For the first null hypothesis––gene therapy is cost-effective––the researcher used only the

data collected from the first question over LPLD. While the price of gene therapy treatments

would inevitably fluctuate depending on different diseases, the researcher concluded his findings

over price with Glybera’s price as the experiment’s baseline. After performing the one-

proportion z-test, the p-value obtained was zero, which means that the proportion of the sample

population that believed gene therapy was cost-effective was significantly less than 50%. As a

result, the researcher must reject the null hypothesis and uphold the alternative hypothesis that

gene therapy is not cost-effective when taking into account a currently available gene therapy

treatment’s price.

For the second null hypothesis––gene therapy is ethical––the researcher performed two

one-proportion z-tests to determine if (1) gene healing and if (2) gene enhancement were ethical.

The p-value in the first experiment was one; the p-value in the second experiment, 9.82 x 10-6.

The first p-value was greater than .05, causing the researcher to retain the null. The second p-

value was less than .05, causing the researcher to reject the null. In other words, the proportion

of the sample that believed gene healing was ethical was significantly greater than 50%, while

the proportion of the sample that believed gene enhancement was ethical was significantly less

than 50%. As a result, in the majority’s opinion, gene healing is ethical while gene enhancement

is not ethical.

GENE THERAPY 30

 

In summary, the first null hypothesis was rejected, leading the researcher to accept the

alternative hypothesis that, in the majority’s opinion, gene therapy is not cost-effective. Next, the

second null hypothesis was divided into two parts: gene healing and gene enhancement. Gene

healing was considered ethical in the majority’s opinion; gene enhancement, unethical in the

majority’s opinion. The researcher then upholds the first null hypothesis for gene healing while

rejects the null hypothesis for gene enhancement, leaving gene healing is ethical but gene

enhancement is not ethical.

Discussion

These findings from the researcher’s survey contributed to the discussion found in the

review of related literature. Again, gene therapy involves the “insertion of a functioning gene

into cells to correct a cellular dysfunction or to provide a new cellular function” (Cotrim &

Baum, 2008). Though gene therapy seems to hold enormous potential, the researcher wished to

discover aspects that may possibly hinder gene therapy from being a worthy pursuit. In

summary, the three final aspects were gene therapy’s risks, the costs, and the ethics. Like

previously stated, the risks would most likely decrease as scientists continue to perfect the gene

therapy procedures. In fact, a simple search on the worldwide web would produce countless

more successes than the year before; in other words, the amount of risks involved in gene

therapy would reduce with time and more research. As a result, the researcher deliberately

focused on finding the majority’s opinion on the last two aspects: cost and ethics.

Findings on Cost

In the review of related literature, the researcher concluded that gene therapy treatments

would be improbable to attain for a common patient in today’s society. Since Glybera was the

first gene therapy treatment on the market, official prices of other gene therapy treatments cannot

GENE THERAPY 31

 

be found. Consequently, the researcher decided to find out whether his sample would pay for

Glybera and then what would be the sample’s ideal price of gene therapy.

Previously, the final decision on Glybera’s price of $1.6 million dollars was that it was

cost prohibitive to anyone except the wealthy. Nevertheless, the researcher’s findings furthered

this statement. With a plurality of the sample earning more than $10,000 a month, the findings

concluded that the majority (89%) would not pay for a $1.6 million dollar treatment. In other

words, even the wealthy individuals would have issues with paying for such an expensive

treatment. As the only alternative to treating this rare disease was to take on a low-fat diet, most

people would prefer this alternative rather than paying the price of Glybera. With that said, the

researcher’s sample follows the trend to criticize the prices of other rare diseases. Originally,

since gene therapy is commonly thought to be permanent (which can be true or untrue depending

on the treatment), the cost could be justified; but according to these findings, this price may still

be out of reach for those with LPDP, wealthy or not (Staff Writer, 2012; Whalen, 2012).

Next, the results of the Glybera question generate another question on what would then

be the majority’s ideal price. The findings produced $1,000 to $9,999 as the leading price range,

with 37% of the vote. Keep in mind, $1,000 to $9,999 is significantly lower than Glybera’s $1.6

million and also the brain atrophy treatment’s $100,000 for the first two years (Lopatto, 2012;

Staff Writer, 2012; Whalen, 2012). As a result, the current prices available for market and trial

gene therapy treatments do not fit the majority’s ideal price range.

Also, in the review of related literature, the researcher discussed intellectual property and

genetically modified organisms (GMOs). Both ideas tie into the findings found in the

researcher’s study. With intellectual property, the researcher found that scientific research

priority lies in caring for the rich rather than the poor. Like previously said, the rich should be

GENE THERAPY 32

 

able to dictate what is being researched and continually obtain intellectual property from the

poor’s cheap labor. Nevertheless, if even the wealthy desires a lower cost for gene therapy

treatments, would not the price decrease? With GMOs, the researcher concluded that research

companies earned the majority of the profits. (Abergel). Applying this idea to gene therapy, the

research companies of gene therapy would naturally profit the most; hence, the price of gene

therapy would probably be expensive.

In the end, the findings concur with what was found in the review of related literature.

With the rejection of the null hypothesis, the findings conclude gene therapy is not cost-effective

or not worth the cost. Likewise, many criticized the price of Glybera; thus, the researcher

considered it safe to deduce that gene therapy treatments would continue to retain its

expensiveness. Will all treatments be around $1.6 million? Probably not as gene therapy

treatments are the future of medicinal.

Findings on Ethics

From the review of related literature, the researcher found that there are two types of gene

therapy treatments: gene healing and gene enhancement. As most individuals would not

question the aspect of healing, many may question the ethics of gene enhancement. Gene

enhancement would bring eugenics to a whole different level, and gene enhancement naturally

becomes the next step after gene healing (Rifkin, 2006). Nevertheless, scientists promise that the

focus would be on “repairing” rather than “replacing”; thus, scientists will postpone the existence

of gene enhancement treatments (Gert, 2001).

All this said, these precautions basically reveal even scientists consider gene

enhancement unethical compared to the less controversial gene healing. The researcher’s survey

aimed to determined what the majority thought about the ethicalness of gene healing and gene

GENE THERAPY 33

 

enhancement. In summary, the gene healing part of the null was retained; the gene enhancement

part was rejected. Thus, according to the majority’s opinion, gene healing is ethical (91%); gene

enhancement is unethical (65%). These results would support the precautions taken by the

scientists. With a mainly Christian-populated sample, the researcher concluded that most likely

the Christian morals and values conflict with a person “replacing” instead of “repairing” one’s

genes. Moreover, the most of the sample can be classified as “rich”; thus, the rich, who are most

likely the only ones able to benefit from gene enhancement treatments, would not agree with

such a procedure.

Despite the researcher presenting two distinct definitions for gene healing versus gene

enhancement, the line between these two types of gene therapy treatments can become obscure.

On the other hand, extremes definitely exist. For example, parents can genetically alter their

babies before birth to obtain super strength, intelligence, resistance to toxins, and many more

(Gert, 2001). On the other hand, gene healing can cure cancer.

Yet, where is the line between the ethical and unethical distinctly drawn? Taking a

previous example stated, one company intended to develop a treatment for hair loss for those

who suffer from chemotherapy (Anderson, 1999). This motive seems genuine and ethical;

however, a gene is added into a person’s genome. Therefore, in the end, this treatment is

classified as gene enhancement. Also, when humans grow old, their hair falls out or becomes

white. This gene would stimulate hair growth or even cause the hair to retain its natural color. A

person could easily argue when a man grows older, genes begin to become faulty; consequently,

these new genes inserted into the body will only repair the faulty ones. If the argument wins,

human beings will continue to look young and never look old; next, human beings will not only

preserve the outside appearance but the inward functions as well.

GENE THERAPY 34

 

In the end, the line separating gene healing and enhancement does not exist, but in the

future, a set location for such a line will most likely be debated frequently. For now, the

researcher’s findings state gene healing is ethical and gene enhancement is unethical.

Implications

Mainly, the researcher conducted the experiment to set parameters according to the

sample’s opinion. In other words, the researcher wishes to inform scientists on the public’s

opinion on the ideal price and potential limitations for gene therapy. These findings established

certain policies such as the ideal price of gene therapy. With a price range from $1,000 to

$9,999, the research companies of gene therapy would most likely achieve success in selling

their products. The second policy concerns the gene therapy’s research practice. Presently, the

researcher’s findings reveal the majority would remain uncomfortable if scientists researched

how to genetically enhance the human body; therefore, scientists should solely focus on gene

healing. Lastly, the findings distinctly lay out the majority’s thoughts on the extremes of gene

therapy; however, the obscure middle area remains untouched. So, a future study on where

exactly is the line between what is ethical and unethical in gene therapy should be conducted.

Conclusion

The researcher findings clearly laid out a portion of the public’s opinion on gene therapy.

While the findings may be biased towards high-income and Christian individuals, the findings do

present information worth considering in future research concerning gene therapy. In the

quantitative research design, the two null hypotheses tested were in the majority’s opinion, gene

therapy is cost-effective and in the majority’s opinion, gene therapy is ethical. The first null

hypothesis was rejected, leading the researcher to accept the alternative to the null hypothesis.

Simply, the majority believed that gene therapy was not cost-effective and would not be a worthy

GENE THERAPY 35

 

pursuit unless the price ranges from $1,000 to $9,999. The second null hypothesis was then split

into two subsections: gene healing and gene enhancement. For the researcher’s findings on

ethics, the researcher did not collect enough evidence against the null hypothesis with gene

healing but did collect enough evidence against the null hypothesis with gene enhancement,

leaving the first part of the null hypothesis retained but the second rejected.

Remaining Questions

All in all, these findings did lead the researcher to further questions. At the crux, gene

therapy is a treatment to pursue perfection through genetics. Then, what is perfection? In terms

of definition, perfection comes from “perficio,”––“to finish” (Lowndes, 2008). As a result,

“perfectio(n)” literally translates to “a finishing” (Lowndes, 2008). Perfection is “in essence, a

being or object in its whole, complete state” (Lowndes, 2008). Socially, this “whole, complete

state” of perfection means a beautiful body on the outside and a healthy body on the inside. In

other words, gene therapy can help one obtain inward and outward “perfection.” Like all good

research, the review of related literature leads the researcher to further questions involving the

concept of immortality, the role of healthcare insurance, the cosmetic industry, the nutritional

industry, and the use of genetic pedigrees or family trees. Nevertheless, the researcher focused

more on the last two subjects for question––the nutritional industry and the use of genetic

pedigrees (or more commonly referred to as family trees). So as a result of researching gene

therapy, the researcher looked into two related questions: (1) how are the nutritional supplements

industries impacting the United States economically today? and (2) how has the benefits of

genetic pedigrees affect the United States socially today?

GENE THERAPY 36

 

Nutritional Supplements

Also, as some people recognize health issues in the United States, they have acted upon

gaining a healthy body. Nonetheless, these actions may not include working out, exercising, or

eating right; instead, why not just pop in a nutritional supplement pill? According from a report

from Packaged Facts Nutritional Supplements in the U.S., this industry’s sales hit $11.5 billion in

2012 and predict sales hitting $15.5 billion by 2017 (Shultz, 2012). For example, the industry

Herbalife has collected revenue of $14 billion and continues to grow at a 5.7% annual growth

rate (“Vitamin & supplement manufacturing…,” 2013). With that statistic, the researcher

echoed the success of the nutritional supplements industry.

Currently, while multivitamins are not doing too well, “conditioned-specific products are

doing especially well” (Shultz, 2012). These products include digestive supplements, Omega-3s

(heart health supplements), joint health supplements, and eye supplements (Shultz, 2012). Joint

health supplements alone posted $140 million in sales (14% increase from the previous year);

eye supplements posted $114 million in sales (9% increase from the previous year) (Shultz,

2012). As clearly seen, many Americans utilized supplements for their ease in maintaining a

healthy body. Furthermore, the consumers over the age of 65 (Baby Boomers) make up the

“backbone” of market support for supplements in the United States (Shultz, 2012). On the other

hand, the supplement usage rates among younger consumers have been declining (Shultz, 2012).

Inevitably, the supplements industry holds a large influence on the United States’

economy. A study from National Products Foundation showed “the dietary supplement industry

to the U.S. economy is [earning] more than three times annual consumer sales, or $61 billion

dollars per year” (“New study shows…,” 2014). Beyond that fact, this industry provides through

production and sales a total of 450,000 jobs (“New study shows…,” 2014). The industry’s

GENE THERAPY 37

 

success makes the dietary supplement industry an important part of the United States economy.

In fact, it is connected to multiple other industries: retail and wholesale trades; real estate, rental,

and leasing; finance and insurance; professional, scientific, and technical services; and

manufacturing (“New study shows…,” 2014). As a result, the dietary supplement industry

becomes interconnected to other industries and ultimately the United States economy. With the

growth of sales in the industry, the extended effects on the economy remain as well, providing

sustainable jobs, manufacturing infrastructure, and health care benefits by way of prevention

(“New study shows…,” 2014). The industry is doing extremely well; even if the economy

becomes unstable, the industry will continue to find success (“New study shows…,” 2014).

The industries of supplements have rooted themselves for success in the future. They

continue to collect a large number of faithful consumers; as a result, their impact on the United

States economy has risen. Supplements now treat a wide range of parts of the body. As people

continue to buy these supplements to obtain a healthy body, this industry has connected itself to

the United States economy, such as providing 450,000 jobs. With the industry booming, the

pursuit of inward perfection can be clearly seen high on people’s priority list.

Genetic Pedigrees

The next remaining question revolves around the uses of genetic pedigrees and their

social implications. Today, genetic pedigrees seem to be a positive result of genetics in action,

showing that gene therapy will most definitely have positive consequences as well. Specifically,

since humans have long known that diseases seem to be passed down from one generation to the

next, the genetic pedigrees can become a powerful “screening tool” that has often been referred

to as the best “genetic test” (“Understanding genetics…,” 2010).

GENE THERAPY 38

 

Often, both common and rare diseases can be found in families (“Understanding

genetics…,” 2010). As a result, family history provides substantial information about a person

past and future (“Understanding genetics…,” 2010). In other words, genetic pedigrees allow

doctors to be able to diagnose at-risk family members for certain diseases. It is important to note

that not all diseases are childhood disorders but can be developed during a patient’s adult years

as well (“Understanding genetics…,” 2010). On the other hand, genetic pedigrees can also

accurately predict which common diseases would not affect a certain family; however, the

absence of a certain genetic disorder in one’s family history should not be rule out completely

(“Understanding genetics…,” 2010). In the end, all this information becomes crucial in the

identification of potential health problems in the future. For example, five to ten percent of all

breast cancer are found to be hereditary (“Understanding genetics…,” 2010). Another example

is Alzheimer’s disease, where in some cases this disease is hereditary (“Understanding

genetics…,” 2010). Therefore, the doctor and patient are able to act upon these potential dangers

to reduce risks by “implementing lifestyle changes and increasing disease surveillance”

(“Understanding genetics…,” 2010).

With family history being such an important tool, the use of pedigrees should be

encouraged and utilized. A pedigree is simply a visual representation of “family members and

relationships using standardized symbols” (“Understanding genetics…,” 2010). Information on

these pedigrees usually include general information such as names and birthdates, family origins

or racial/ethnic backgrounds, health status, ages of deaths and causes of death for each family

member, and pregnancy outcomes (“Understanding genetics…,” 2010). With such information,

genetic pedigrees should be updated more often in hopes to being able to avoid future problems

with genetic disorders or diseases.

GENE THERAPY 39

 

Nowadays, family trees have many uses, including the possibility of connecting with

unknown family members or the discovering one’s ethnicity. Nevertheless, pedigrees with

genetic information take the uses of pedigrees to another level. Pedigrees have the ability to

indirectly preserve life and health of patients. Sites, such as ancestry.com, have come to this

same realization and have offer the ability to form pedigrees with “advance DNA science” at the

cost of $99. Even with such a cost, people should consider forming their family pedigrees,

taking into account all the benefits that come with these simple family trees.

Biblical Connection

After considering how gene therapy connects to the world today, the researcher

concludes gene enhancement is not worth the cost when compared to the present consequences

of pursuing the ever-changing status of “perfection.” Even so, where does one draw the line in

enhancing the body? On the other hand, humans are still required to take care of their

appearance and sustain personal hygiene and even health. As seen through the use of genetics in

pedigrees, not all gene therapy treatments are necessarily “bad.” Nonetheless, in Psalms 139:13,

David writes, “For you formed my inward parts; you knitted me together in my mother's womb.”

In this verse, humans must acknowledge the work of God, who created humans individually and

uniquely. If scientists were to genetically alter God’s work permanently, this act defeats the

whole purpose of God creating each human being for His eternal good will. In other words,

humans now control their own genes, which was once surrendered to God’s will. While having

control of one’s own genes should be discouraged, the change is not plainly stated in the Bible to

be a sin. If it were a sin, one can argue that dyeing one’s hair color is a sin also. In approach to

this argument, 1 Peter 3:3 states, “Do not let your adorning be external–the braiding of hair and

the putting on of gold jewelry, or the clothing you wear.” In other words, God commands

GENE THERAPY 40

 

humans not to be consumed with the external. If humans are too focused on glorifying

themselves, they will miss the purpose of glorifying the God that truly deserves praise and

worship and glory. Therefore, when one considers genetic enhancements, one must ask the

question: What is the purpose of getting this gene replaced and whom would it glorify? In fact,

this question applies to many decisions today, but man remains confused on what his priorities

are. At the end of the day, the main priority is definitely the perfection found from within,

mentioned in 1 Samuel 16:7 with how “the Lord looks on the heart.” So, God desires virtuous

men; men that seek to glorify God rather than themselves. David was a man after God’s own

heart. While even David was not sinless and therefore not perfect, his heart was the Lord’s. He

pursued perfection in terms of a righteous life more than he sought outward perfection. Thus, if

gene therapy is ever to be utilized, it must lie in the boundaries of healing instead of

enhancement.

GENE THERAPY 41

 

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http://www.clinmedres.org/content/4/3/218.long

GENE THERAPY 42

 

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the-costs

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B%20Releases&%20refno=181&view=DSIB_Releases_Detail

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GENE THERAPY 43

 

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food.html?pagewanted=all

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ENGET0001&SingleRecord=True.

GENE THERAPY 44

 

Appendix

Copy of Survey

Hi, this is Kevin Chin. I am currently enrolled in a course known as Distinguished

Scholars Program (DSP), which allows students to research a topic of their interest. Having

researched Gene Therapy for my junior year, I now hold the task of collecting data for this

coming year. Please consider taking this survey for my research. Furthermore, if you would

share this link with others, I would be extremely grateful. Your response will remain anonymous.

Finally, taking this survey indicates your consent for your response to be used in my research.

Thanks again for your time.

1. Age

2. Ethnicity

a. Caucasian

b. African-American

c. Hispanic

d. Filipino

e. Asian or Pacific Islander

f. American Indian or Alaskan Native

g. Other

3. Occupation

a. Arts, Entertainment, & Sports

b. Business

c. Health and Medicine

d. Media and Social Sciences

GENE THERAPY 45

 

e. Public and Social Services

f. Science, Math, and Technology

g. Trades and Personal Services

h. Other

4. Personal Monthly Income

a. Less than $1,000

b. $1,000 - $4,999

c. $5,000 - $10,000

d. More than $10,000

5. Estimate the Average Amount Spent on Medical Treatments Annually in the Last 3

Years

a. Less than $1,000

b. $1,000 - $4,999

c. $5,000 - $10,000

d. More than $10,000

6. Religion

a. Christian

b. Jewish

c. Muslim

d. Hindu

e. Buddhist

f. Non-religious

g. Other

GENE THERAPY 46

 

7. Would you pay the price of $1.6 million dollars if a close family member had LPLD?

Glybera is the only gene therapy treatment on the European (not on American)

market. Currently, it costs $1.6 million dollars. A major contributor to the

development of Glybera, Dutch Company UniQuire, justifies this price by the simple

fact that Glybera reaches a small market of lipoprotein lipase deficiency (LPDP)

victims. LPLD causes a person to be inable to process fat; thus, the victim's blood has

high levels of fat, which causes adominal pain. Currently, a way to treat LPLD is to

eat a low-fat diet.

a. Yes

b. No

8. What would be your ideal price range for genetic therapy treatments?

Answer with comparisons to the cost of past medical procedures

a. Less than $100

b. $100 - $999

c. $1,000 - $9,999

d. $10,000 - $49,999

e. $50,000 - $99,999

f. $100,000 - $999,999

g. $1,000,000 - $2,000,000

h. More than $2,000,000

9. Is genetically healing a person ethical?

Genetic healing - the changing or replacing of a faulty gene to heal a disease

a. Yes

GENE THERAPY 47

 

b. No

10. Is gene enhancement ethical?

Gene enhancement - the adding or changing traits to the human body (could range

from changing hair color to becoming like Captain America)

a. Yes

b. No

Copy of Survey Results

Age (List of ages found below)

35 36 34 39 37 38 43 42 40 22 24 25 26 27 30 17 18 15 16 70 59 58 57 56 55 65 62 60 61 49 48

45 44 300,000 47 46 51 52 53 54 50

Ethnicity

Caucasian 144 72%

African-American 13 7%

Hispanic 9 5%

Filipino 2 1%

Asian or Pacific Islander 23 12%

American Indian or Alaskan Native 1 1%

Other 8 4%

Occupation

GENE THERAPY 48

 

Arts, Entertainment, & Sports 6 3%

Business 34 18%

Health and Medicine 22 12%

Media and Social Sciences 3 2%

Public and Social Services 16 9%

Science, Math, and Technology 22 12%

Trades and Personal Services 12 6%

Other 71 38%

Personal Monthly Income

Less than $1,000 25 13%

$1,000 - $4,999 51 27%

$5,000 - $10,000 26 14%

More than $10,000 86 46%

Estimate the Average Amount Spent on Medical Treatments Annually in the Last 3 Years

Less than $1,000 62 32%

$1,000 - $4,999 72 38%

$5,000 - $10,000 36 19%

More than $10,000 22 11%

GENE THERAPY 49

 

Religion

Christian 182 93%

Jewish 0 0%

Muslim 0 0%

Hindu 0 0%

Buddhist 0 0%

Non-religious 7 4%

Other 6 3%

Would you pay the price of $1.6 million dollars if a close family member had LPLD?

Yes 21 11%

No 171 89%

What would be your ideal price range for genetic therapy treatments?

Less than $100 12 6%

$100 - $999 41 21%

$1,000 - $9,999 70 37%

$10,000 - $49,999 42 22%

$50,000 - $99,999 20 10%

$100,000 - $999,999 5 3%

$1,000,000 - $2,000,000 0 0%

GENE THERAPY 50

 

More than $2,000,000 1 1%

Is genetically healing a person ethical?

Yes 175 91%

No 17 9%

Is gene enhancement ethical?

Yes 66 35%

No 125 65%

Number of daily responses