This document discusses gene therapy, including what it is, how it works, types of gene therapy, benefits and limitations. Gene therapy uses genetic engineering to insert a normal gene to replace an abnormal gene causing disease. The main types are somatic and germ-line therapy. Benefits include treating previously incurable diseases like cancer. Limitations include the short-lived nature of changes and immune responses. The document also discusses ethical, political and social issues around gene therapy research and applications.

1. Ji Yoon Kim
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2. Therapy through Genetics - Gene Therapy
Gene is a unit of heredity that is transferred from a parent to offspring and is
held to determine some characteristic of offspring. That means genes are the one
that chooses the characteristic of the child. Diseases such as cancers are caused
when mutations occur in the gene, it could be congenital and it could be acquired.
These diseases were incurable, however, science has improved rapidly over the
twenty years and is still improving, which allowed humans to cure such diseases.
The therapy that allows people to cure these diseases is called the gene therapy. In
this essay it will talk about what Gene therapy is, its benefits and limitation, ethical
and political issues of Gene Therapy.
The basis of Gene therapy is genetic engineering, which modifies the gene.
Gene therapy use genetic engineering to cure or treat a disease caused by mutations
in the gene. (Dowshen, 2007) In gene therapy, a “normal” gene is inserted into the
humans’ genome to replace the
“abnormal” gene. Vectors are carrier
molecule are used to delivers the
therapeutic gene to the patient’s
abnormal cells (see figure 1). The
most common vector currently is
virus that has been genetically
modified to carry “normal” human
DNA. There are many different types
of viruses that are used as vectors in
gene therapy. An example of a virus
is adenovirus, which is a class of
virus with double-stranded DNA Figure 1- How gene therapy works using adenovirus
genome and causes respiratory, Vector (How Does Gene Therapy Work?, 2010)
intestinal and eye infection in humans, it also causes common colds. (Gene Therapy,
2009)
There are two types of gene therapy, one is Somatic gene therapy and the
other is Germ-line gene therapy. Somatic gene therapy involves introducing a
“normal” gene into the targeted cells to treat the patient. However the genes will not
be passed on to the future children. In other words, even a patient altered their
genes to treat the disease, it won’t change the fact that the disease will be passed on
to the patient’s children. Germ-line gene therapy involves modifying the genes in
egg or sperm cells. In contrast to somatic gene therapy, germ-line gene therapy can
pass any genetic changes to the future generation. However this type of therapy is
still at its infancy and more research is needed. (Barthoshesky, 2010)

3. One of the benefits of a gene therapy is the treatment of an incurable disease.
There are many diseases that human cannot cure, such as diabetes, Alzheimer’s
disease, Parkinson’s disease,
cystic fibrosis and cancer.
Cancer is caused by out of
control cell growth. When cell
division is out of control, cells
divide continuously. This forms
tumours, which is a cancer. In
gene therapy a viral vector is
inserted into the patient’s body
and it allows correcting the
mutation that has happen
during the cell division process
(see figure 2). Gene therapy
gives the opportunity of living a
normal life again to the patient.
(Rocholl, 2010)
The second benefit of a
gene therapy is the prevention
Figure 2 - How to cure Cancer using gene therapy (Gimson,
2006) of congenital diseases. Germ-
line gene therapy, which
involves manipulation of the gene in the egg or the sperm cells, allows passing on
genetic changes to the future generation. If a patient receive a germ-line gene
therapy which cure or treats a certain disease, this gene could be passed on to the
future generation and can have low chance of getting the disease or immune to the
disease. Germ-line gene therapy could help the patient to cure one’s disease and
cure the disease for the patient’s future generation. Germ-line gene therapy could
allow the prevention of congenital diseases, and prevent children with severe
combined immunodeficiency, giving the opportunity of healthier future generation.
(Noguchi, 2003)
Despite of these beneficial aspects of gene therapy there are also many
limitation of the gene therapy. The first limitation of the gene therapy is the short-
lived nature of a gene therapy. For a gene therapy to become a success a therapeutic
DNA introduced into the target cell must remain functional, and the DNA must be
long-lived and stable. There are problems with integrating therapeutic DNA into the
patient’s genome and the rapidly dividing nature of many cells prevent the gene
therapy from achieving long-term benefits. Due to the nature of short-lived gene
therapy, the patient will have to undergo multiple rounds of gene therapy to take an
effect. (Gene Therapy, 2009)
The second limitation is due to the immune system of a human. Whenever a
foreign object is introduced into the human tissues, the immune systems attack the
invaders. The immune systems are designed to attack the invaders and their
responses are enhanced if they have seen it before. Since the common vectors used
for the gene therapy are viral vectors, there is a potential risk of stimulating the

4. immune system, which may reduce the effectiveness of a gene therapy. Stimulating
the immune system not only reduce the effectiveness, it makes it difficult for
repetition of the therapy in patients because of the immune systems’ characteristic.
(Gene Therapy, 2009)
The third limitation of a gene therapy is the problem with the use of viral
vectors. Viral vectors are viruses that are genetically modified carry “normal”
human DNA. However this doesn’t change the fact that they are viruses. The viral
vector, which is meant to help cure the disease inversely, it could cause toxicity,
immune and inflammatory responses, gene control issues. In addition to these
issues, there is always the fear of causing diseases due to the recovery of ability to
cause disease once the viral vector is inside the patient. (Gene Therapy, 2009)
One of the major limitations of gene therapy is the limitation of curing
mutations in a single gene. Gene therapy is the best candidate for the diseases, or
disorders that arose from mutations in a single gene. For some of the most
commonly occurring disorders, such as heart disease, high blood, pressure,
Alzheimer’s disease, arthritis, and diabetes are caused by combined effect of
variation in many genes. It is difficult to use the process of gene therapy to cure or
treat the multi-gene or multi-factorial disorders. (Gene Therapy, 2009)
There are many ethical concerns related to gene therapy. The major ethical
issue with gene therapy, which all gene-relating researches confront, is the issue of
playing god. Eighty years ago, there was a resolution submitted to the U.S Senate.
The people believed that altering the body given by god or the Mother Nature is
wrong and very sinful. These people said that it is unethical to play around with
Mother Nature and what she has created is something sacred and it should not be
changed for any reasons. In 1944, 58% of the people polled by Time/CNN think,
that altering human genes is against the will of God. (Elmer-Dewitt, 1994) There are
still many people believing the Mother Nature as the omniscient and omnipotent
being and humanity should not rise up against it. (Bergeson, 2010)
For a gene therapy research to happen, there are some issues with the
governments. For a gene therapy trial or a protocol to take place, it must be
approved by at least two review boards at the scientists’ institution. After being
approved by the two review boards, it must be approved by the U.S Food and Drug
Administration (FDA). The FDA is in charge of regulating all gene therapy products.
In addition, trials that are funded by the National Institutes of Health (NIH), federal
focal point for biomedical research in the U.S, must be registered with the NIH
Recombinant DNA Advisory Committee (RAC). These steps have to be followed in
order for a gene therapy research to take place. These steps make scientists to take
special precaution with gene therapy. (Gene Therapy for Cancer, 2006)
There are issues with the use of the gene therapy that may lead to social
issues, discrimination. The original use of gene therapy is for the use of curing
diseases caused by genetic disorders, however, proactive citizens for genetic
engineering believe it is possible for gene therapy to help children be smarter, jump
higher, grow taller and furthermore. Problems can arise when parental love goes to
the extreme of making the children as superior as possible. There is also a

5. possibility abuse of this technology. The therapy could be used for cosmetic
correction such as altering skin color, enhancing physical attractiveness and
intelligence. This could cause the discrimination between the rich and the poor.
(Ethical Considerations, 2010)
Gene therapy an outcome of an advancement of science technology may help
people to cure diseases caused by genetic disorders. Despite of its current limitation
of technology, it is one of the promising therapies in the future. Science technology
is instructive unless it is abused. Although gene therapy is a promising therapy, it is
still at its infancy and for it to be widely used for the treatment more researches are
needed.
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