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2014
Gene Therapy Evolution: How Gene Therapy has
Evolved Preventive Medicine
Kayuri Shah
Virginia Commonwealth University
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Gene Therapy Evolution
How Gene Therapy has Evolved Preventive Medicine
Kayuri Shah, Virginia Commonwealth University
Introduction
Gene therapy is defined as a regenerative therapy that contains an active
substance containing recombinant nucleic acid administered to human beings
with the intention of regulating, repairing, replacing, adding, or deleting a genetic
sequence. The goal of gene therapy is to have a therapeutic or diagnostic effect
that relates directly to the desired gene expression. The techniques required for
gene therapy can help several areas that have gone untreated, such as skeletal
diseases, brain tumors, and hereditary diseases. The main focus over the years
has been on bone regeneration avenues through gene therapy. Though gene
therapy has suffered several setbacks during clinical trials due to errors within
human trials, with the support of public research institutions, the media, and the
public, research has picked up the pace. However, how much has gene therapy
helped in the advancement of preventive medicine and bone regeneration?
Gene therapy researchers have yet to define gene therapies’ areas of
applications. Applications range from ex vitro and in vivo, to directly altering the
gene in utero. The ethical and safety concerns are vast if the procedure is used
improperly, and cannot proceed before a line is defined between actual physical
hindrances and cosmic enhancement, meaning the difference between treating
dwarfism and an undesirable height. Without the ability to perform the proper
clinical trials, researchers argue there will be no furthering of gene therapy to
actual human application on a wide market, available to everyone. Despite the
ethical and safety concerns, in order to advance preventive medicine in bone
regeneration, gene therapy protocols need to reach the clinical level, but new
regulations will not allow that before testing is completed on larger animals.
Since there are several methods, such as ex vivo and in vivo, each able to use
viral and non-viral vectors, preventive medicine can improve through the use of
gene therapy.
Evolution of Gene Therapy
Future Goals
Figure 2. Through
the various
pathways of the
human body, there
are numerous
ways that the
different types of
gene therapy
methods could be
used for bone
regenration. Only
this does not show
where the usage
will stop if not
regulated. In utero
and embryonic
development is a
very likely
possibility if even
some methods
were okayed.
Timeline of Therapeutic Events
Various Gene Therapy Methods
In vivo viral vectors
 Vector technology: high
efficiency by viruses
transferring genetic
information into host cell
 Overexpression of genes lead
to faster healing
 Tested in rat models
In vivo non-viral vectors
 Less costly
 Safer
 Minimal immune response
from host
Ex vivo viral vectors (traditional, cellmediated)
 Outside host’s body; foreign
DNA is manipulated with
target cells outside the body
 Tested in large animals
 Can be used in combination
with several types of vectors
Ex vivo non-viral vectors (expedited,
cell-mediated)
 Technically combining ex vivo
and in vivo methods
 Least experimented with, yet
most likely to be used in an
operating room
References
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(2012). Gene therapy for brain tumors: Basic developments and clinical
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(2012). Gene therapy approaches to regenerating bone, Advanced Drug
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1. What regulations will be required to ensure the
safe usage of gene therapy”
2. Before clinical trials can be reached, what are the
next step for researchers to take so the methods can
start being applied to humans again, after several
setbacks?
3. What factors determine the best use of the
therapeutic methods?
4. How far along is the rest of the world in this
research?
Conclusion
Preventive medicine has reached a long way through
gene therapy, definitely helping the science behind bone
formation. However, the largest obstacle that is still
presented is the safety concerns surrounding each
method, and without testing on larger animal models,
there is no way to know how the therapy products would
react on a human. The pitfall of regenerative medicine is
that it can only be done on live models, and therefore,
must also be carefully monitored in its usage.
Regardless of the setbacks in gene therapy, the
regulations that have been set in place by the US FDA,
suggested by the NIH and other researchers, have
allowed for safer clinical trials. Not only do we want to
prevent deaths, but the misuse of the methodology.
Gene therapy has yet to be completely defined in its
purpose; no line has been drawn between using the
products for cosmic reasons, or using it for actual
genetic conditions. Neither has anyone explored the
possibilities of what may happen if gene therapy is done
in utero, and the implications on the mother, baby, and
society. Current legislation only allows gene therapy to
be conducted in somatic cells, but that does not mean
there will not be those that attempt to perform it in
gamete cells as well. However, in many other countries,
gene therapy products are already on the market, such
as Europe and China, and therefore, in the United
States, it is necessary that we progress to the next level,
in order to bring this medicine to the public in a safe, yet
quick and efficient manner.
Osawa, K., Okubo, Y., Nakao, K., Koyama, N., Bessho, K. (2010).
Osteoinduction by repeat plasmid injection of human bone morphogenetic
protein-2, Journal of Gene Medicine 12, (937-944). Retrieved from Wiley
Online Library:
http://onlinelibrary.wiley.com.proxy.library.vcu.edu/doi/10.1002/jgm.1515/pdf
Wirth, T., Parker, N., Ylä-Herttuala, S. (2013). History of gene therapy. Gene 525
(162-169). Retrieved from Science Direct:
http://www.sciencedirect.com.proxy.library.vcu.edu/science/article/pii/S0378
111913004344
Zallen, D. (2000). US gene therapy in crisis, Genetics and Society 16, 272-275.
Retrieved from Science Direct:
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952500020254#