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Is it ethical to use gene therapy to cure genetic disorders?
Courtesy of slimfilms.com
Mercer University School of Engineering
EGR 108 Professional Practices
Dr. Marjorie Davis
J. Ryans
The Bubble Boy
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In 2002, then 18month-old Rhys
Evans received one
of the first
successful gene
therapy treatments
for a genetic
disorder, which
cured his severe
combined
immunodeficiency
(SCID).
Courtesy of news.bbc.co.uk
Common Terminology
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Adenovirus- Originally a harmful virus filled with doublestranded DNA that can be modified into a transport vessel for
inserting therapeutic genes.
DNA- A very long molecule that carries a cell's genetic
information.
Gene- A segment of DNA found on a chromosome that
codes for a particular protein.
Genome- The sum of all genes that code for a particular
organism.
Vector- Gene therapy delivery vehicles, or carriers, that
encapsulate therapeutic genes for delivery to cells. These
include both genetically disabled viruses such as adenovirus
and non-viral vectors such as liposomes.
The Gene Therapy Process
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A desired gene is selected and
inserted into a genome to replace
an “abnormal”, disease-causing
gene.
The genome is then transferred
into a vector, most commonly an
adenovirus, which will transport
the gene into the patient.
The vector is sent to a target cell
in the patient’s genome and then
“unloads” the genetic material in
the virus.
Once the genetic material has
been inserted into the target cell,
the cell will continue its
reproductive cycle with the new
therapeutic gene . This will cause
new generations of cells with the
integral therapeutic gene which
will cure the desired disease in the
patient.
Application of Gene Therapy
One of the primary uses for
gene therapy at its current stage
is to cure particular genetic
disorders. These include
hemophilia, chronic
granulomatus disorder, and
severe combined immune
deficiency. In the future gene
therapy will hopefully be able to
cure other types of acquired
diseases. Such as
neurodegenerative diseases,
hepatitis, and even cancer.
Courtesy of news.bbc.co.uk
Major Advancements
2002- Scientists are able cure children
of SCID.
 2003- Researchers are able to insert
genes to the brain, opening up a wider
variety of diseases that could be cured.
 2006- Scientists successfully treat two
cancer patients.
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Problems with Gene Therapy
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Difficult to integrate desired DNA into a
genome.
Effects are usually temporary so patients
need multiple rounds of treatment.
Immune system automatically attacks
foreign objects in the body, which can
reduce effects of gene therapy.
The possibility of the modified adenovirus
to revert back to its harmful state.
In rare cases therapy has induced tumors,
specifically leukemia.
Economics of Gene Therapy
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Courtesy of dir.nichd.nih.gov
Funding for therapy
fluctuates with rise
and fall of success.
Most funding in
government research,
averaging $400
million a year.
Extremely difficult for
most of society to
afford and can’t be
covered by insurance.
Ethical Issues
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Who decides what
diseases to cure?
Who will have access
to therapy?
Is it interfering with
God’s plan?
Will it bring about
negative eugenics?
Is it alright to use the
therapy in the prenatal
stage of development
in babies?
Courtesy of www.cbs.dtu.dk
Conclusion
Gene therapy has the capability of curing
numerous, common diseases in the future.
 Therapy can also have severe, unforeseen
circumstances.
 At its current stage, therapy not accessible
to most of society due to cost.
 The ethical issues of gene therapy greatly
slow its progress, but most are important to
consider before expanding the
development of the technology.
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Annotated Bibliography
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Baum, C., Schambach, A., Modlich, U., & Thrasher, A. (2007). [Gene therapy of SCID-X1]. Medline,
RetrievedMarch 15, 2008, from
http://www.ncbi.nlm.nih.gov/pubmed/18046520?ordinalpos=13&itool=EntrezSystem2.PEntrez.
Pubmed.Pubmed_ResultsPanel.Pubmed_RVDocSum
This source was an article that gave technical insights to how a common genetic disease works and
ways for gene therapy to combat it. It’s also a highly reliable source because the paper come from a
top government research institute.
Maugh, Thomas (2007). Gene Therapy. In Microsoft® Encarta® Online Encyclopedia [Web]. Microsoft
Corporation. Retrieved March 15, 2008, from
http://encarta.msn.com/encyclopedia_761579447_3/Gene_Therapy.html
This source provided mostly general information of how gene therapy works and the possible
applications of it. This source also provided very good media components to help convey the idea of
gene therapy.
Terminology. Retrieved March 15, 2008, from American Society of Gene Therapy Web site:
http://www.asgt.org/about_gene_therapy/terminology.php
This source was helpful in providing terms I could tell my audience prior to explaining gene therapy.
For such a technical subject it would be almost impossible to accurately explain the concept without
knowing certain terms.
Hoag, H (2005, May, 26). Gene therapy rising? Nature, 435, Retrieved March 15, 2008, from
http://www.nature.com/nature/journal/v435/n7041/full/nj7041-530a.html
This article gives opinions and statistics about the economics of gene therapy. This is important
because it gives possible options for people in the future for accessing gene therapy and who it will be
accessible to.