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Gene therapy is a technique that uses genes to treat or prevent disease. Gene therapy replaces a mutated gene that causes disease with a healthy copy of the gene. Introducing a new gene into the body helps fight a disease (U.S department of health and human services (2019)-gene therapy. Retrieved from ghr.nlm.nih.gov). Gene therapy uses viruses to deliver DNA in viral vectors. Inside the viral vectors are a healthy gene to replace the mutated gene. Gene therapy isn’t officially available to the public but has been tested on individuals with genetic diseases. Although gene therapy is a promising treatment for many diseases including inherited disorders, types of cancers, and viral infections. The treatment remains risky and is still being studied to make sure it’s effective and safe for the patient being treated. However, in March 2006, researchers announced the successful use of gene therapy to treat two individuals for X-linked Chronic granulomatous disease. This study is the first to show that gene therapy is successful when treating the myeloid system. ‘
Chronic Granulomatous Disease (CGD) is an inherited primary immunodeficiency disease which increases the body’s susceptibility to fight infections that are caused by bacteria and fungi (American Academy of Allergy, A.(updated2019)-CGD. Retrieved from aaaai.org) Granulomas are large amounts of immune cells that form at the site of an infection or inflammation. Chronic Granulomatous Disease is a rare disease and only affects 1:250,000 births (Nowicki, R. J. (2018, April 30)-Chronic Granulomatous disease. Retrieved from emdeicine.medscape.com). Patients with chronic granulomatous disease are not able to fight off common germs. This leads to the patient being very ill from common infections that are usually mild in healthy individuals. This is because the chronic granulomatous disease makes it very difficult for cells named neutrophils to produce hydrogen peroxide. The Immune system needs hydrogen peroxide to fight off many kinds of bacteria and fungi. Severe infections can include skin, bone and abscesses in internal organs such as lungs, liver or brain (American Academy of Allergy, A. (updated 2019)-CGD. Retrieved from aaaai.org). Apart from the defective neutrophil function in Chronic granulomatous disease, the rest of the immune system is normal. Individuals with chronic granulomatous disease can be healthy until they become infected with a germ. Children can be born with chronic granulomatous disease and be healthy at birth, but later develop infections in infancy or early childhood.
But gene therapy hasn’t been successful in other cases for example in 1999, gene therapy suffered a major setback when 18-year-old Jesse Gelsinger died during gene therapy. Jesse Gelsinger was doing a gene therapy trial for ornithine trans carboxylase deficiency (OTCD). OTCD is a genetic disease that causes too much ammonia to accumulate in the blood. Jesse died from multiple organ failures 4 days into the gene therapy trial. After examination, his death is believed to have been caused by a severe immune response to the adenovirus carrier (Karen Norrgard, P. (2008)-gene therapy. Retrieved from www.nature.com). Although the treatment sounds promising there are still a few risks. Some of these risks include: The immune system may respond to the working gene copy that has been inserted by causing inflammation, and the working gene may be put in the wrong spot.
The basic steps of gene therapy include:
A benefit of gene therapy on chronic granulomatous disease is that gene therapy provides a short term relief for a patient suffering this disease. This short term relief is created from one gene therapy treatment. If a patient wants long term relief they would need to undergo repeated treatments of gene therapy.
A limitation of gene therapy on chronic granulomatous disease is that gene therapy has not become a permanent treatment for this genetic disease. Because the DNA introduced into the cells must remain functional and the cells containing the therapeutic DNA must be a permanent and stable treatment (sources, m. (2019)-Gene Therapy. Retrieved from en. Wikipedia). But DNA into the genome and the rapidly dividing cells prevent gene therapy on becoming a successful permanent treatment on patients.
Gene therapy has the ability to replace defective cells in chronic granulomatous disease. This means that the chronic granulomatous disease after repeated gene therapy could potentially be cured. But repeated gene therapy can lead to other altercations (mentioned in limitations).
Immune response: Normally a foreign object that is introduced into human tissue, the immune system will straight away attack the potential invader. The risk of stimulating the immune system in a way will reduce the effectiveness of gene therapy is a potential risk (sources, m. (2019)-Gene Therapy. Retrieved from en. wikipedia). The immune system enhanced response to invaders it has seen before makes it extremely difficult for gene therapy to be repeated in patients.
If researched further it has the potential to eradicate chronic granulomatous disease completely in the future. Gene therapy is a life changing treatment which needs to be researched and developed further because of its altercations it holds. Gene therapy can not only create long term relief for chronic granulomatous disease but many other genetic diseases.
A limitation of gene therapy on chronic granulomatous disease is that gene therapy could damage the gene pool. It is believed that gene therapy could possibly change the human gene pool permanently if it is done to a certain degree (Health, U. N. (2018)-Gene therapy for chronic granulomatous disease. Retrieved from clinicaltrials.gov)
A benefit of gene therapy on chronic granulomatous disease, is it gives an individual a second chance of living a normal life. Chronic granulomatous disease can cause a lot of pain and sickness because of the bodies inability to fight infection. This means that an individual suffering from this can constantly be ill with infection.
Potential to giver rise in other disorders: There is an exact point in the patient genome where the right genes should be brought in. While there is no assurance that the viral enzymes used in the process can bring in the correct gene at such point. When things go wrong during the gene therapy treatment it could lead to severe disorders to develop in the patient being treated.
The first ethical issue of gene therapy is the cost. The cost of gene therapy is $500,000-$1.5 million dollars, which would not be able to be purchased by an average citizen (Moschos, S. (2018, April 2)- GENE THERAPY IS NOW AVAILABLE, BUT COULD COST MILLIONS OVER A LIFETIME, SAYS SCIENTISTS. Retrieved from www.independent.co.uk). The gene therapy cost restricts ordinary people to getting the treatment meaning a lot of people would be missing out on a life changing treatment. About 1,000-2,000 total people in the U.S could be able to afford the treatment meaning not many Australians can afford this treatment. Marrazo from scientific America admits that $400 million dollars have been spent of gene therapy (Hirschler, B. (n.d.)- Gene Therapy is now available but who will pay for it. Retrieved from www.scientificamerican.com).Patients who want to receive gene therapy won’t pay the large bill upfront but spread the treatment cost over months. If the price stays the same for gene therapy a large amount of people who really need the treatment won’t be able to be treated for their harmful genetic disease. This is a huge disadvantage for people who are not able to afford the price. This means that thousands of people will have to continue to suffer because of the price it is meant to retail at on the market (when available).
The second ethical issue is the danger of testing gene therapy on humans. Gene therapy holds many risks to patients during the clinical trial. Gene therapy uses viruses to deliver DNA in viral vectors. The viral vectors are then inserted into the body. But there is a risk that the DNA could be introduced into the patient’s reproductive cells (N.A. (2019)-Risk Factors. Retrieved from www.genetherapynet.com). This could produce changes that may be passed on if a patient has child after the treatment. This is a very dangerous risk because it could lead to the patient’s baby developing even harmful mutations in the womb or infancy.
The second risk is if the gene is inserted in the wrong location in the DNA, it could eventually lead to harmful mutations to the DNA or even cancer. This has happened to 4 out of 20 patients in a clinical trial for x-linked severe combined immunodeficiency disease (N.A. (2019)- Risk Factors. Retrieved from www.genetherapynet.com). X-linked SCID is an inherited disorder that occurs in only males. This makes these individuals prone to reoccurring infections. This is because they lack the necessary immune cells to fight certain bacteria’s (Medicine, U. N. (2019, February 12)-X-linked severe combined immunodeficiency. Retrieved from ghr.nlm.nih.gov). Because it was inserted in the wrong spot it led to 4 patients developing T cell leukemia.
T cell leukemia is the overproduction of abnormal or immature white blood cells that circulate or are present in the blood. T cell leukemia is a rare but often aggressive disease with a median survival rate of 10.2 months Tobinai, k. (2009, 16 December)-Current Management of Adult T-Cell Leukemia/Lymphoma. Retrieved from www.cancernetwork.com). In this example it shows that the scientists didn’t have enough knowledge on where the defective cell was. This mistake led to patients developing a life threatening disease, that has no return. This shows that maybe they aren’t as knowledgeable when it comes to treating patients. “Director of the Pittsburgh Human Gene Therapy Center and President of the American Society of Gene Therapy Joe Glorioso said that gene therapy for immunodeficiency through manipulation of bone marrow still looks ‘exceedingly promising” (Gene Therapy volume 9, pages 1561–1562 (2002)).
In conclusion, both these ethical issues effect the progress of gene therapy being a successful and permanent treatment in the future.
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