Benefits + Limitations replace defective cells or even eradicate certain

diseases. All these points work in favour of the

In all sickle cell anemia cases, i find all ways of
gene therapy but if we take a further look, we
treatment very important as they are some of the
notice many moral issues that may arise. Once
only ways sickle cell anemia can be treated. A
gene therapy is being used in humans, how can we
arge benefit is that with gene therapy, doctors are
make sure it is only being used for medical
able to fix mutated genes which hasn’t ever been
purposes instead of enhancing human capabilities?
done efficiently before so this would be a huge
We can’t have people using gene therapy to make
scientific breakthrough. Another benefit is that
their kids smarter or stronger, it needs to be used
gene therapy is able to make diseased cells
to save lives. We also know that gene therapy is a
evident for the immune system so it actually helps
very modern cure and is still in research but when it
your body naturally fight sickness. Although there
comes out, it will be very expensive. How will it be
are many important benefits, limitations will always
distributed in a way that it can be accessed by
exist, the first one is that once the virus returns to
people who need it, instead of only the wealthy?
your body during gene therapy, it can return to its
These are all ethical topics that are constantly
original strength and may even become worse.
being debated as the advantages and
Gene therapy has also been proven to cause
disadvantages of treatment. I believe, if used in the
tumors if inserted in the wrong part of our DNA so
right way and for the right reasons, gene therapy
hat is a very serious consequence. In my opinion,
can work wonders in saving lives and families.
f scientists could somehow breakthrough in finding
a cure through gene therapy, many people with the
disease would be helped. In conclusion i believe
Sources
that science does a reasonable job in dealing with
problem but it can definitely be improved by finding “Sickle Cell Disease.” National Heart Lung and Blood
Institute, U.S. Department of Health and Human
echniques that are not harmful for the patient such
Services, www.nhlbi.nih.gov/health-topics/sickle-cell-
as gene therapy which is the safest option.
disease.
Scientists are headed in the right direction, all that
s left is doctors being successful with gene therapy “Sickle Cell Anemia.” Mayo Clinic, Mayo Foundation for
on a person without a high risk of negative side Medical Education and Research, 8 Mar. 2018,
effects. www.mayoclinic.org/diseases-conditions/sickle-cell-
Moral Implications
Although research in gene therapy is very important
anemia/diagnosis-treatment/drc-20355882.
Sickle Cell Anemia
important, there are many ethical and controversial “Sickle Cell Disease - Genetics Home Reference - NIH.”
dilemmas that seem to arise. Advantages of these U.S. National Library of Medicine, National Institutes of
Health, ghr.nlm.nih.gov/condition/sickle-cell-disease.
treatment methods are obvious as people are
treated of sickle cell anemia and their immune
“What Are the Ethical Issues Surrounding Gene Therapy?
system is reinforced by bone marrow transplant. - Genetics Home Reference - NIH.” U.S. National Library
For gene therapy, it’s advantages are perhaps even of Medicine, National Institutes of Health,
GARVEY KNAPP                     FRIDAY OCTOBER 5TH
LEVEL III BIOLOGY                              2018
5 more important as they have the ability to ghr.nlm.nih.gov/primer/therapy/ethics. 6 1
 What is sickle cell
Genetic Causes
anemia+Problem
For my project i have a patient that was diagnosed
with sickle cell anemia. The father of this child has Sickle cell disease is caused by a mutation in the
already been diagnosed with sickle cell anemia and hemoglobin-beta gene found on the 11th
it is possible that the mother is a carrier of a mutated chromosome. It will be called the hemoglobin S
hemoglobin S gene. This child was diagnosed at gene that can cause blockage and slowing of blood
birth and has been coming in frequently with feelings flow, which will reduce oxygen and this can cause
of a low energy level, episodes of pain and we have severe episodes of pain. A child that is born with
noticed lung tissue damage which is an indicator of sickle cell disease has inherited this genetic
sickle cell anemia due to the difficult passage of red disorder from a carrier of the mutated gene who is

blood cells and the lack of oxygen.
First we must ask, what is sickle cell anemia? Sickle fact is that someone who carries the sickle cell trait
cell disease is a form of anemia which will be
inherited at conception, by a single pair of genes,
one from each parent. It is a recessive pattern of
inheritance, therefore you must have two copies of
the sickle cell allele in order to have sickle cell
anemia. Sickle cell anemia is when the human body
does not have enough healthy red blood cells. This
will result in an insufficient amount of oxygen being
brought to the body. The “average” person’s blood
cells are round as well as flexible which will allow
them to flow smoothly through our blood vessels,
but, with sickle cell anemia, red blood vessels are
shaped like crescent moons and are rigid, this will
make it much more difficult for them to flow smoothly
through our blood vessels. So, blood cells have a
iron rich protein called hemoglobin which helps
deliver oxygen, for someone with sickle cell anemia,
this hemoglobin is abnormal. As well as having a
rigid shape, sickle cells last up to 20 days whereas
normal red blood cells can live up to 120 days. This
means that they burst much earlier and red blood
cell count is lower than normal. Sickle cell anemia is
a very frequent disorder which 7500 americans are
affected by. The disease is most frequent among
african americans and hispanic americans. 2
Solution
either their mom and or their dad. An interesting
will not always get the disease but, they can easily A very scientific way researchers are looking into
pass it down to their children. treating sickle cell anemia is through a process
called gene therapy. By using gene therapy,
researchers look to correct the defective genes
and insert it into the patients bone marrow. This
will encourage the production of normal
hemoglobin. For a simple explanation, what
researchers have done to this day in relation to
gene therapy is tested it on mice. Scientists were
successful in creating mice with the human
defective gene that causes hemoglobin S.
Scientists removed bone marrow from the mice
and were able to correct the hemoglobin S gene
with the beta-hemoglobin which they hoped would
“Sickle Cell Disease.” National Heart Lung and Blood Institute,
U.S. Department of Health and Human Services, produce more red blood cells. This bone marrow
www.nhlbi.nih.gov/health-topics/sickle-cell-disease. with the “corrected” genes was then transplanted
into the mouse who had sickle cell disease.
Above, I have attached a picture of 2 parents with Subsequently, the mouse started producing much
sickle cell trait as they have the mutated higher levels of red blood cells. Although that it is
hemoglobin S gene as well as a normal very important that this worked on a mouse,
hemoglobin A gene. People with sickle cell trait scientists are still a long way from being able to
are mostly healthy but with a mutated hemoglobin conduct this procedure for a human. Scientists are
S gene from each parent, you will have sickle cell hoping to start testing this on humans to see how
anemia (4th child). far or close they are to making this seemingly
3 perfect treatment a reality. 4