Issues Related to Gene Therapy

This article discusses the advancements in gene therapy as a potential treatment for genetic diseases. It explains how gene therapy works, its potential risks, and the regulations that should be put in place to prevent abuse of the technology. The article also provides an example of how gene therapy can be used to treat sickle cell disease.

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ISSUES RELATED TO GENE THERAPY.
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Abstract.
According to (Explore Gene Therapy 2018), genes direct the body to make vital proteins for
various functions. The genes come in pairs, one from each parent. Faulty or missing genes
trigger genetic diseases (Explore Gene Therapy 2018). Gene therapy seeks to curb monogenetic
diseases; caused by a single faulty or missing gene pair. The therapy gives the body a new
working pair of new gene that is either missing or faulty in order to facilitate protein synthesis.
Discovered in the early 1970`s, the gene therapy has shown positive results in correcting genetic
disorders (Explore Gene Therapy 2018). In 2003, China approved the treatment for neck and
head cancers using gene therapy and in 2017, the therapy was approved for treating genetic
disorders causing blindness (Explore Gene Therapy 2018). This discovery is therefore a great
advancement in medicine and treatment of genetic disorders.

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ISSUES RELATED TO GENE THERAPY.
Gene therapy has been studied as a potential treatment for genetic disease since the
1980’s; the goal of gene therapy is to help cure genetic diseases by providing a functioning copy
of the gene to make up for the genetic defect. A functioning copy of the gene is packaged into a
lentiviral vector (adeno-associated virus); this is a modified virus that acts as a transport vehicle
for the functioning gene. The virus is used as the vector because of its ability to enter the body
and moreover, it is not known to cause any diseases (Explore Gene Therapy 2018). The lentiviral
vector delivers the functioning copy of the gene into the nucleus of stem cells that have been
taken out of the patient, the gene is then inserted into the DNA of these cells and is thereafter
able to direct the body on how to make the protein required, thus curbing the effect of the genetic
defect. In recent years, scientists have made improvements in these vectors and continue to study
gene therapy as a treatment for many different diseases including cancers, blood diseases, central
nervous system disorders, and immune system diseases.
https://www.exploregenetherapy.com/about-gene-replacement-therapy
Gene therapy is a new technology which menus it is costly, and only affordable by
higher class (The Economist, 2016). It can be used for genetic modification and if not regulated,
genetic therapy could be abused by the general public who are looking for physical enhancement
or even cosmetic improvements. Nations may also use this to create enhanced and unstoppable
armies (Future of Working n.d.). Therefore, in order to avoid this, a group of highly trained
scientists should cooperate with world governments, together deciding who is fit to be treated
under gene therapy and what type of gene abnormalities should be cured though genetic therapy.

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In addition, regulations on the extent on which the therapy should be used for physical
enhancement should be set in order to prevent formation of enhanced unstoppable armies that
may result in wars.
An example of a genetic disease that is worthy of genetic therapy is sickle cell disease
(SCD). SCD is an inherited disorder; affecting millions of people around the world. Screening
for sickle cell disease is typically done at birth, patients with sickle cell disease have abnormal
hemoglobin. Hemoglobin is the protein in red blood cells that delivers oxygen to all cells in the
body (Niihara, 2013). The abnormal hemoglobin causes the red blood cells to become ridged and
take on a sickle or a crescent shape, the sickle red blood cells get stuck in the body’s small red
vessels, as a result, cells and organs cannot get the oxygen they need leading to organ damage
and failure, as well as episodes of pain known as crisis. The most common treatment for sickle
cell disease includes antibiotics, pain management, IV fluid, blood transfusion, hydroxyurea and
surgery (Berliner & Parise, 2015). Transfusions deliver healthy red blood cells to the body,
however, they also cause buildup of extra iron in the body causing organ damage, abdominal
pain, weakness and fatigue. Patients who receive ongoing transfusions must also take additional
medicines to remove the iron buildup (Pietrangelo, 2013). This is why gene therapy has been
introduced as a new treatment for sickle cell disease (Fox, 2015). A functioning copy of the
hemoglobin gene is packaged into a lentiviral vector, the vector contains a small number of parts
from HIV, which is very effective at moving genes into cells, and the vector has been modified
so it cannot cause HIV infection (Fox, 2015). The patient’s blood stem cells will be collected
from a bone marrow harvest procedure; the blood stem cells are separated from liquid bone
marrow. Using the lentiviral vector, the functioning copy will be inserted into the DNA of the
collected stem’s blood cells (NH, 2001). Inside the body, chemotherapy will be used to remove
existing stem cells to make space for the new ones. The modified blood stem cells will be
returned to the patient’s body where they are expected to grow and produce new cells that
contain normal hemoglobin. The goal of third treatment is for the modified stem cells to become
a permanent source of healthy red blood cells that do not sickle, preventing organ damage and
other complications (CIRMTV, 2015)]
In conclusion, gene therapy is a great advancement in technology, it shows how
the human race has evolved to create new techniques that have influenced the lives of many and
has contributed to saving many individuals from their fate. Gene therapy gives a second chance

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to those who were not lucky enough and have had no say in the problems caused by their birth
simply because, of the family they were born into and the DNA that they were born to possess.
However, the use of gene therapy contributes too many unfavorable things such as an
overpopulated Earth, the lowering of the death rate in the human population, the increase in our
population size, meaning, more pollution and less resources.

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References.
Berliner, N., & Parise, L. V. (2015, December 28). Sickle Cell Disease: Challenges and Progress.
Website Online. Available from, http://www.bloodjournal.org/content/127/7/789
CIRMTV. (2015). Defeating Sickle Cell Disease with Stem Cells Gene Therapy. Website Online.
Available from, https://www.youtube.com/watch?v=f3pjyQvBdz0
Fox, C. (2015). New Sickle Cell Gene Therapy Approaches. Website Online. Available from,
http://www.dddmag.com/article/2015/04/new-sickle-cell-gene-therapy-approaches
NIH. (2001). Scientists Use Gene Therapy to Correct Sickle Cell Disease in Mice. Website
Online. Available from, https://www.nhlbi.nih.gov/news/press-releases/2001/scientists-
use-gene-therapy-to-correct-sickle-cell-disease-in-mice
Niihara, Y. (2013). Understanding Sickle Cell Disease. Website Online. Available from,
http://www.news-medical.net/health/Understanding-Sickle-Cell-Disease190613.aspx
Pietrangelo, A. (2013). Transfusion Therapy. Website Online. Available from,
http://www.healthline.com/health/transfusion-therapy
The Economist. (2016). Why gene-therapy drugs are so expensive. Website Online. Available
from, http://www.economist.com/blogs/economist-explains/2016/08/economist-explains-
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Explore Gene Therapy. (2018). Gene Replacement Therapy. Website Online. Available
from, https://www.exploregenetherapy.com/about-gene-replacement-therapy
Future of Working. (n.d.). 6 Advantages and Disadvantages of Gene Therapy. Website
Online. Available from, https://futureofworking.com/6-advantages-and-disadvantages-of-gene-
therapy/