Saini 1
Tarlin Saini
Ms. Gaspar
SBI 3U1
10 April 2015
Gene Therapy Potential
Gene therapy is an experimental treatment that involves introducing genetic material into
a person’s cells to fight or prevent disease. A number of diseases are being researched, such as
Hemophilia, Parkinson’s disease, cancer and even HIV, through various approaches. A vector is
used to deliver a gene to a cell, most commonly viruses. They can recognize certain cells and
carry genetic material into the cells' genes. (Mayoclinic.org) The viruses that are used in gene
therapy, are altered to make them safe, but can still carry some risks. The gene therapy
technology is still in its infancy, but it has been used with some success and is expected to
improve in the future. (Genetherapynet.com)
There are two different kinds of gene therapy treatment: somatic cell gene therapy and
germline therapy. The somatic gene therapy includes treating just the faulty genes by replacing
them with the correct genes. By doing so, the body will then produce the accurate proteins
needed to eliminate the gene causing the disease. This procedure merely corrects the recipient’s
genes but cannot be passed down to his or her offspring, unfortunately. With the germline
therapy, however, it is possible to pass the corrected genes down to the next generation. This is
done by having the gene inserted into the reproductive cells, so that the gene can be inherited by
any future offspring. (Chu et al.)
There are several ways of fighting off diseases with gene therapy, which include fixing
the mutated genes, or making diseased cells more evident to the immune system or simply
replacing the mutated genes. Fixing the mutated genes involves the turning off of mutated genes,
if possible, and the turning on of healthy genes that prevent the disease, so that they can inhibit
the disease. In other cases, the immune system may not attack diseased cells because it doesn’t
recognize them as intruders. Gene therapy can be used by doctors to train the immune system to
distinguish those cells as threats. On the other hand, simply exchanging the genes that are
Saini 2
mutated, as they cannot correctly or at all, can help treat diseases. For instance, p53 is a gene that
normally prevents tumor growth in your body. This p53 gene has been linked to several types of
cancer, but if doctors could substitute the defective p53 gene, the cancer cells have the possibility
to be triggered to die. (Mayoclinic.org)
Although gene therapy has it’s potentials it carries its risks as well. Genes cannot be
inserted directly into cells, rather they have to be delivered using a carrier, called a vector. The
risks include, unwanted immune system reactions, wrong targeted cells, infections caused by the
virus, and possibilities of causing tumors. Unwanted immune system reactions, being one of the
risks, is when our body's immune system may see the newly introduced viruses as intruders and
attack them. This may cause inflammation and, in severe cases, organ failure. Another risk
includes, targeting the wrong cells; because viruses can affect more than one type of cells, it's
possible that the altered viruses may infect additional cells — not just the targeted cells
containing mutated genes. If this happens, healthy cells may be damaged, causing other illness or
diseases, including cancer. You can also get an infection caused by the virus; it’s possible that
once introduced into the body, the viruses may recover their original ability to cause disease.
Last but not least, there is a possibility of triggering a tumor; if the new genes get inserted in the
incorrect spot in your DNA, there is a chance that the insertion could possibly lead to tumor
formation. This has occurred occasionally in some clinical trials. (Mayoclinic.org)
There are many things that can go wrong with gene therapy, but it definitely seems like
it’s worth the shot, if it has the ability to even lessen the disease outcomes, much less cure the
disease. Gene therapy can cause modifications and alterations to human capabilities, which can
change the future for humans for good. Along with that, it can also damage gene pools
permanently, also considering only the rich would be able to afford such a treatment. There can
also be a rise in disorders with gene therapies. While the most common choice as a vector, using
the virus for such a purpose is not always guaranteed. There’s an exact point in the host genome
where the right gene should be brought in and there is no assurance that the viral enzyme
responsible for this step will be able to bring in the correct genes at the exact point in the host
genome. If there’s an inaccuracy in the process, the consequences could bring about severe
disorders. (OccupyTheory)
Saini 3
Gene therapy has the capacity to better fight, and avert diseases involving genetics. It is
the lone treatment for diseases involving genetics, as it can replace defective cells with suitable
ones. It has had quite a lot of accomplishment in the past. In the case of Hemophilia, the disease
involves blood clots, where large sums of blood can be lost through internal bleeding. In a small
trial, researchers successfully used an adeno-associated viral vector to deliver a gene for Factor
IX, the missing clotting protein, to liver cells. After treatment, most of the patients made at least
some Factor IX, and they had fewer bleeding incidents. This then proves that gene therapy had
the ability to make the disease less severe, which is a step forward for gene therapy.
(Learn.genetics.utah.edu)
The world of gene therapy seems to hold a lot of promise, but to this day, several barriers
stand between gene therapy, such as ethical reasons, discovering a reliable way to get genetic
material into cells, reducing the side effects, and the aim to target the precise cells. It may have
its barriers but it has shown some success in treating diseases such as, severe combined immune
deficiency, Hemophilia, blindness caused by retinitis pigmentosa and as well as Leukemia.
(Mayoclinic.org)
Currently gene therapy stands as a possible and very promising opportunity to cure
genetic disorders, by utilizing the introduction of accurate genes into an incorrect patient’s
genome. However, scientists have been in motion with gene therapy since the 1990s and
although they have made progress by treating diseases such as Hemophilia B, they aren’t yet
convinced it is eligible nor safe to be introduced to the public. Researchers are currently working
on finding a non-viral vector to carry the gene as a safer supply of the corrected gene. If
successful, this will limit the side effects and fearful consequences of gene therapy as well as
make it minimize the risk level for a patient. Personally, I believe future goals of gene therapy
will involve allowing the opportunity of gene therapy to more individuals and countries wanting
to experiment in curing their genetic disease, and the progression of ethic, and social which
surround the process, and the reduction of social implications which follow. In addition, future
steps to be taken include the acceptance of government officials to agree to advance funding for
the study and experiments of gene therapy to gain progressive knowledge on the topic. In whole,
Saini 4
future steps for gene therapy entail much more research in perfecting the practice, and making it
more socially aware and accepted in a consistently changing society. (Rubanyi)
On the topic of the future for gene therapy, there are many jobs out there for scientists
that study genetics. For example, Geneticists make between $30,000 and $55,000 a year, and
genetic researchers generally earn between $50,000 and $100,000 a year, although some earn
more. Pfizer Inc. is a company that focuses on gene therapy research, and there are jobs open for
scientists as well, which is a great opportunity, and therefore fortifies the future for gene therapy.
(Www2.careercruising.com)
There may be a lot of work to do on the theory of gene therapy, and there are many
arguments on the cons, and ethical issues. Although there is much controversy, most agree this
treatment can bring a bright future for humans therapeutically. In conclusion, the potential of
gene therapy does outweigh the risks, but in a long-term scale. (The Crux)
Saini 5
References
Chu, Joyce et al. 'Gene Therapy Benefits And Potential Risks'. Guardian Liberty Voice. N.p.,
2015. Web. 9 Apr. 2015.
Genetherapynet.com,. 'Gene Therapy Jobs'. N.p., 2015. Web. 9 Apr. 2015.
Genetherapynet.com,. 'Gene Therapy Technology Explanied'. N.p., 2015. Web. 9 Apr. 2015.
Inc, Pfizer et al. 'Gene Therapy Jobs, Employment|Indeed.Com'. Indeed.com. N.p., 2015. Web. 9
Apr. 2015.
Learn.genetics.utah.edu,. 'Gene Therapy Successes'. N.p., 2015. Web. 9 Apr. 2015.
Learn.genetics.utah.edu,. 'What Is Gene Therapy?'. N.p., 2015. Web. 9 Apr. 2015.
Mayoclinic.org,. 'Gene Therapy Results - Mayo Clinic'. N.p., 2015. Web. 9 Apr. 2015.
Mayoclinic.org,. 'Gene Therapy Risks - Mayo Clinic'. N.p., 2015. Web. 9 Apr. 2015.
Mayoclinic.org,. 'Gene Therapy Why It's Done - Mayo Clinic'. N.p., 2015. Web. 9 Apr. 2015.
OccupyTheory,. 'List Of Pros And Cons Of Gene Therapy'. N.p., 2015. Web. 9 Apr. 2015.
Rubanyi, Gabor. The Future Of Human Gene Therapy. 1st ed. Richmond, CA: N.p., 2015. Print.
The Crux,. 'With Gene Therapy We Could Direct Our Own Evolution'. N.p., 2015. Web. 9 Apr.
2015.
Www2.careercruising.com,. 'Career Cruising'. N.p., 2015. Web. 9 Apr. 2015.