Running head: THE ROLE OF GENE THERAPY IN MEDICINE 1

The Role of gene Therapy in medicine

Damian Martinez
Bayside High School








THE ROLE OF GENE THERAPY IN MEDICINE 2
Abstract
This paper will discuss gene therapy and how it has developed through out the years, as well as
to explain the role of gene therapy in the medical field. This paper will cover, discuss, and
provide information for past trials that where preformed including the first trial in 1990 that dealt
with a 4-year-old girl with an immune system deficiency (Blease, R. (2014)). Another topic that
will be covered is an explanation of how mutations and disorders develop in someone and gene
therapy works to help cure it. Also information on the two different types of gene therapy
explaining the difference among the two plus the many vectors that go along with inserting the
gene into the desired target will be provided with information by Blaese, R. (2014) and other
sources.
Although many successes go along with gene therapy there are also concerns and flaws that
follow. This paper will explain both the scientific and social concerns with the fairly new
practice of gene therapy. Finally we will look at how gene therapy could influence future
medical practice as well as see what can be improved upon this form of treatment using insight
from Trefil, J. (1996)

Keywords: gene therapy, deficiency, vectors, development, treatment, scientific,
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The Role of Gene Therapy in Medicine
Imagine living in a world where if someone developed cancer all they had to do is get a
shot at the doctors office. As easy as that, just to cure a very serious disease, well this could be a
reality thanks to gene therapy. Gene therapy is a fairly new technique that helps by inserting
normal or healthy genes to correct disorders. According to Blaese, R. (2014) the first
treatments were given in 1990 and since that, scientists have wanted to figure out its hidden
potential such as its possible aid in curing incurable hereditary diseases. Therefore gene therapy
has and will play a big role in in the development of future medical practices.
Gene Therapy
Mutations play a role in why people develop disorders and hereditary diseases and a
mutation occurs when a section of the DNA is coded wrong and it could be in many different
ways like duplication of one chromosome or a deleted chromosome (Vink, in class lecture,
2014). This is where gene therapy plays its role in helping to cure these diseases. According to
Blaese, R. (2014).Gene therapy is an experimental technique for treating or preventing diseases
by inserting a gene into a patient's cells. Genes are the basic unit of heredity. They carry the
chemical instructions that determine the form and function of each cell (Blaese, R.(2014).
Through gene therapy, doctors can provide a new set of instructions for treated cells. Once the
gene is inserted into the body the carrier makes its way to the target area and places the gene and
soon it gets rid of the unwanted gene (Gene Therapy (2014). So gene therapy may seem simple
through this explanation however there are many other options to choose such as the correct type
of therapy or what type of gene should be use.
Types of therapy
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Gene therapy is a vast and confusing topic however scientists have determined the two
types of gene therapy currently possible and those are somatic gene therapy and reproductive
gene therapy also referred to as germline gene therapy (Trefil, J. (1996). (Somatic gene therapy
is therapy that is performed on the somatic cells also known as body cells. This therapy is
currently in use and it works by inserting the treated gene into a body cell usually in the bone
marrow or the targeted site of the mutation and it inserts the fixed cell into the cell to fix the
mutation. The second type is reproductive gene therapy and according to Blaese, R (2014) it is
different to somatic in that it now involves the reproductive cell such as the sperm and ova.
Although this type of therapy is still theoretical it could treat hereditary diseases that are pass
down from parent to child such as Down syndrome. Blaese, R (2014) said that reproductive gene
therapy works exactly as somatic gene therapy, so once the gene is inserted into the sperm or ova
the mutation is fixed and it is them passed on to the offspring of that person. Today gene therapy
has come a long a long way since its discovery and there have been many trials that have been
performed to get to where we are now.
Past trials
From the first trial in 1990 to now there have been many trials to help develop a better
understanding for topic of gene therapy and not much would be know if it werent for the past
trials and the research conducted. However with great success come unfortunate disasters and
setbacks. Information from Gene Therapy, (2014) states that the first gene therapy trial ever
conducted occurred in 1990 at the National institute of health in Bethesda, Maryland and it
involved a 4-year-old girl with an ADA (adenosine deaminase) deficiency and ADA is important
to the immune system. So the doctors in Maryland decided to try gene therapy by taking some of
her genes and fixing the mutated section of DNA and it was successful (Gene Therapy, (2014).
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However, not all past trials were like the first one, many failed by either the patient dying or
developing some other disease such as the French trial of 2000. Levine, L (2014) reports that
in the trial of 2000, French scientists tried a different treatment for ten infants that had an
immune system deficiency. However unlike the trial of 1990 four of the ten infants developed
leukemia and other scientists claimed that it was due to the gene therapy that they received. From
then up to 30 gene therapy trials were canceled and this was a disadvantage to the further
development of gene therapy during that time. Since a lot of trials have failed in the past people
developed many scientific and social concerns about this new practice.
Concerns of gene therapy
With the development of any new treatment come concerns from people of how safe a
new treatment is and gene therapy is no exception. Levine, L. (2014) agrees that many
concerns with gene therapy arise. For example, because gene therapy is still fairly new to the
field of medicine not many facts are known about gene therapy so there are still unknown factors
that go with the treatment. Also according to Gene therapy (2004) there are also many
problems with vectors. Vectors are what carry the enhanced gene to the target so that it can be
fixed in the body (Gene therapy (2004). The problem with the vectors is that some are not
precise such as liposome vectors; others have the possibility of reactivating and causing harm
such as disabled virus vectors. Finally there are also many social concerns with gene therapy
because many people think that the genes that someone is born with shouldnt be changed and
this is a major reason for why gene therapy isnt a top priority to research ( Hunt, S. (n.d.).
Unfortunately there are still many problems with gene therapy, but in the future with enough
development and research it could really help people.
Future of gene therapy
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The perfect usage of gene therapy is far from close to obtain; however the future is bright for
gene therapy. Gene therapy has many opportunities to be successful because of the way the
treatment is delivered, through easy injections and according to Trefil, J. (1996) the diseases
that gene therapy will cure first are the widespread diseases such as cancer. Another
improvement that could possibly happen in the future that concerns medicine is that more
research could go into gene therapy such as the research of new vectors (Gene therapy (2004).
For example adenovirus vectors and aden-associative virus vectors could become the way of
delivering the treatment in the future because they provide a safe and precise way of delivering
the treatment. Finally gene therapy has the possibility of curing currently incurable diseases such
as cancer and cystic fibrosis.
Closing
Today gene therapy could open the door to new techniques and procedures that could possibly
cure disorders. With more research and development of vectors, carriers, and new ways of
delivery, gene therapy could become an easy and effective procedure. Although there have been
some setbacks and unfortunate disasters, it is possible that sometime in the near future if
someone develops a life threatening condition, gene therapy could be their answer.













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References
Blaese, R. (2014). Gene therapy. In World Book Student. Retrieved from
Gene therapy (2004). The Hutchinson Dictionary of Ideas. Retrieved from http://sks.sirs.com
Gene Therapy. (2014). The New Book of Knowledge. Retrieved January 13, 2014, from Grolier
Online http://nbk.grolier.com/ncpage?tn=/encyc/article.html&id=10005065& type=0ta
Hunt, S. (n.d.). Controversies in Treatment Approaches: Gene Therapy, IVF, Stem Cells, and
Pharmacogenomics. Nature.com. Retrieved January 11, 2014, from
http://www.nature.com/scitable/topicpage/controversies-in-treatment-approaches-gene-
therapy-ivf-792
Levine, L. (2014). Gene Therapy. Grolier Multimedia Encyclopedia. Retrieved January 12,
2014, from Grolier Online http://gme.grolier.com/article?assetid=0117655-0
Trefil, J. (1996). Is there a future for gene therapy? 101 Things You Don't Know About Science
and No One Else Does Either. Retrieved from http://sks.sirs.com