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Ryan Kato
Professor Collin Hull
English 2010
6 June 2012
Gene Therapy
Cure for humanity or Night of the Living Dead?
Gene therapy is the insertion of genes into an individual’s cells and tissues to treat a
disease in which a mutant allele is replaced with a functional one. Imagine if you could prevent
your children from having disorders such as cancer, Cystic Fibrosis, or Parkinson’s, but you may
also damage your future gene pool; what would you do?
Discussion:
Scientist and politicians have long argued about the study of genes (biological or
natural) and the moral affect it will have on our culture. The party against gene therapy argues,
“Gene therapy has the potential to be misused – for instance the concept of ‘designer babies’
where specific genes are selected in order to create the perfect child, can be compared to
Hitler’s attempts to create a superior race” (“Pros,” Par 12). The party for gene therapy
argues, “If gene therapy targets the reproductive cells of carriers of such genetic disorders like
Cystic Fibrosis, Parkinson’s disease, or cancer, it is possible that any children the carrier goes on
to have would be free of the defective gene and on a bigger scale the disease can be wiped out
completely” (“Pros,” Par 3)? Both parties have a good understanding of each other and the
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effects gene therapy could have on the medical field, and a biological evolution. As we examine
the current state of gene therapy we discover there are still a lot of unanswered questions. The
Food and Drug Administration (FDA) has pointed this out in the following statement;
The food and drug Administration (FDA) has not approved any gene therapy
product for commercial sell. Current gene therapy is experimental and has not
proven very successful in the United States. Little progress has been made since
one of the first gene therapy clinical trials begun in 1999. In 1999, gene therapy
suffered a major setback with the death of 18-year-old Jesse Gelsinger. Jesse
was participating in a gene therapy trial for Ornithine Transcarboxyase
Deficiency (OTCD). He died from multiple organ failures four days after starting
the treatment. His death is believed to have been triggered by a severe immune
response to the adenovirus carrier (“Gene,” Par 6).
From this we can see there is great potential to solve a lot of diseases that plague mankind, but
there is also a great risk of opening Pandora’s Box.
History:
To better understand genetics let’s review the
history. Gregor Mendel, an Austrian priest born in 1822
was the first acknowledged person to study the field of
genetics. In the 18th century Mendel described how
traits are inherited from generation to generation. He
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also demonstrated that inherited genetic traits could be modified to affect a different genetic
outcome. In the 1970’s the idea was floated in several scientific communities that “gene
surgery” could be used to treat several genetic disorders. Dr. Richard Mulligan, the professor of
genetics at Harvard University forwarded the idea that viruses could be ideal vectors for
introducing modified genes into the human body (“Genetics” Par 2). The benefits of gene
therapy were proven successful in the case of Ashi DiSivla;
In 1990, a 4-year-old girl named Ashi DiSilva was the first patient to receive gene
therapy for Severe Combined Immunodeficiency (SCID). Ashi’s parents were
willing to face the unknown risks to their daughter because they were already far
too familiar with the risks of SCID-the couple’s two other children also had SCID
and were severely disabled. Ashi is now a healthy adult with an immune system
that is able to fight off most infections (Belk and Maier, pp.217).
The successful outcome of Ashi’s case proved the benefits that gene therapy can provide for
Immunodeficiency’s. Ashi’s case
also, sparked interest to scientist
and biologist all over the world to
further understand what gene
therapy is and what it can do for us.
We now have gone from proving a
single case to a whole product line
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of developed drugs.
“China in 2004 following 14 years of development launched the first commercial
product of gene therapy for the treatment of head and neck squamous cell
carcinoma (skin cancer). Called Gendicine, which used an Adenoviral Vector
injected into the affected area that would bind to the tumor cells and destroy
the affected gene. Following the launch they did a study on 120 patients, with
no patient relapse observed over 3 years,” (“Pharmaprojects.com,” 2012).
Following China’s breakthrough, around the world several breakthroughs have led to several
patents in drugs. These include Collatagene the treatment for Limb Ischemia and Buergers
Disease, Advexin which is similar to Gendicine with neck and shoulder treatment, and Cerepo
which treats Glioma a type of brain cancer (“Pharmaprojects,” 2012).
Risks:
To be fair to the science of gene therapy we also have to view the failures and the
potential risk involved with genetic manipulation. As mentioned earlier, the largest setback in
gene therapy research happened in 1999, when Jesse Gelsinger died during his gene therapy
trial. Shortly after his death the National Institutes of Health (NIH) reported that there had been
hundreds of failed gene therapy experiments including a number of deaths (“BrightHub,” 2012).
Additionally the moral and ethical consequences of gene therapy are coming into focus.
More and more people are getting concerned about changing someone’s naturally inherited
genes thus playing the role of God. “The moral challenges of gene therapy lead to the question
of whether the procedure is intended to correct a genetic defect in the embryo, or to improve
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the genetic potential of the embryo by supplementing its existing genetic complement with
genes that provide some beneficial trait, such as enhanced intelligence, height, or health
(genetic enhancement)” (Human, par. 6). If man develops the ability to selectively choose the
attributes of their offspring –is this playing God? Is this ethical, and is this fare? Undeniably,
the law of unattended consequences will come into effect. With continuing to push the limits
of gene therapy, do we run the risk of starting a ethical war, or do we simply run the risk of
opening Pandora’s Box to the flesh eating-blood hungry-brainless zombies?
Conclusion
When comparing the benefits and the risk, we can now see just how far gene therapy
has advanced. It started, off in a faraway vineyard with Mendel genetically modifying pea
plants, to what’s now a substantial product line to treat a cornucopia of disorders. Conversely
we can see that this experimentation has led to the failure and the death of test subjects. But
the potential of gene therapy is incredible for what it can offer us in the future.
The potential for mankind in gene therapy studies solemnly outweighs the pitfalls of
changing our moral values and destroying humanity as we know it. Gene therapy has helped
countless people who otherwise would have suffered and died from many different types of
genetic disorders and disease. However like any adventure into the unknown, uncontrolled
manipulation of genes can lead to grave consequences; such as the feared “Zombie outbreak”.
Clearly, sound research and ethical guidelines must be enforced in order to control the
outcome, especially the negative outcome. There should be little remaining question of the
current benefits and issues that have been explored, however the choice is up to you; Is gene
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therapy and its quest to end disease as we know it, worth the potential unknown risks that
Pandora’s Box could cripple us with?