• If you are citizen of an European Union member nation, you may not use this service unless you are at least 16 years old.

  • You already know Dokkio is an AI-powered assistant to organize & manage your digital files & messages. Very soon, Dokkio will support Outlook as well as One Drive. Check it out today!


Paul's Gene Therapy Evaluposal

Page history last edited by Paul Elden 12 years, 4 months ago

A nice long read...





     Death is a sad, yet unavoidable part of life. Everyone has experienced, or will experience, the death of a loved one. It is a rough thing to go through. Often times it is felt that the deceased was whisked away too soon, or that they had more to live for. These thoughts could someday become preventable, as the diseases or conditions that affected the deceased become preventable. This may be achieved through gene therapy. For people with genetic disorders or acquired conditions such as cancer, the chance to live a full, complete lifetime is closer than most think. Gene therapy is in its early stages, but has already helped dozens of people with illnesses that were previously thought incurable. Gene therapy is a good medical treatment technique because it could prevent hereditary illness and disease, eventually eradicate such diseases, and allow individuals that would be born with hereditary diseases lead better, more normal lives.


     There are two different types of gene therapy: somatic and germline. Somatic gene therapy is quite ingenious, and has two different forms. During in-the-body gene therapy, bioengineers modify an existing virus, usually an adeno-associated virus. This is a type of virus that is not known to cause any disease in humans. The engineers then add in synthesized DNA designed to produce a specific reaction within cells. The virus is injected into the patient, and it attacks the patient’s somatic cells (non-sex cells). These cells have specific genes removed by the virus, and the virus then implants the synthetic genes into the host cell’s DNA. The faulty genes have now been removed, and the proper genes in place.  This cell will start acting as it should, then multiply and replace the dysfunctional cells. In addition, the virus will reproduce, causing faster spreading of the proper genetic code. In-the-body gene therapy is the choice for patients with hemophilia, as adeno-associated viruses attack the liver and blood first. This can effectively spread non-hemophilia genes quickly to areas associated with it. (Arthur Neinhuis)


     Out-of-the-body somatic therapy is different, but effective in its own way. Doctors take either blood or bone marrow from the patient, and then isolate immature cells. In these cells it is easier to modify their own DNA, as opposed to adding new DNA, due to their early developmental stage. The cells are placed back into the patient’s bloodstream, and make their way back to the bone marrow (where blood cells are manufactured). These fixed cells replicate and edge-out damaged cells, and correct the deficiency or disorder of the patient. Out-of-the-body therapy can also be achieved through the use of retroviruses; HIV is an example of a retrovirus. Retroviruses are highly specialized in replacing host DNA, so genes are easily implanted into the patient. However, retroviruses are also able to place their genes anywhere in the host DNA, which could possibly damage the patient even further. This treatment has been very successful in treating Severe Combined Immunodeficiency. Children with SCID have no immune system, and can die from the most insignificant virus or bacteria. Among those treated for SCID, gene therapy has been successful in over 90% of cases; however, five children developed leukemia, but four have been cleared of it. Doctors are now in the process of refining delivery systems in order to place genes in the correct places, and somatic gene therapy looks very promising. (Arthur Neinhuis)


     Human germline genetic modification (or HGGM) is a highly controversial treatment that has not yet been attempted. In HGGM, a patient introduces specific genes or a specific gene into either their eggs or sperm. This type of gene therapy is meant to permanently alter future generations’ DNA. In most cases, this would be used to prevent the presence of hereditary disease, like cystic fibrosis, in offspring. (Genetics & Public Policy Center)


     In the case of HGGM, it would be possible to prevent and even completely eliminate hereditary conditions like Tay-Sachs disease or hemophilia. Genetically-passed diseases such as these are contained within the genes of the parents. The parents may not be affected, but the genes could be passed on and activated in the offspring. Future parents may be able to undergo HGGM and erase the disease-causing genes from their gametes, or eggs and sperm. This would allow the offspring to be born free of the possibility of having an inborn condition. Not only would this technique be able to prevent the disease, it could eliminate it all together if the DNA specific to certain conditions disappeared over time. Tens of thousands more children would be born healthy and normal each year.


     Patients that undergo somatic gene therapy can also lead much happier and more normal lives. In 2007, doctors led by Philippe Leboulch of Harvard Medical School used an engineered virus to insert a section of DNA into an unnamed patient with Beta-thalassaemia. This disease is a type of anemia that affects the blood’s ability to carry oxygen, leading to possible organ failure and death without regular blood transfusions. As of June 2008, the man received his first transfusion in over a year, and was reported to be in good health. (Giuliani Ferrari) Success like this is what drives gene therapy science forward.


     Of course, this experiment is just one of many attempts at gene therapy. These trials come with limited and widely varied amounts of success. Many of the arguments against gene therapy would ask if the risk is worth the reward. The most famous failure with gene therapy is the story of JesseGelsinger. Gelsinger was born with OTC deficiency. This results in the inability to break down ammonia, a natural byproduct of metabolism, and leads to death in about fifty percent of cases. In 1999, Gelsinger, only 18, underwent a gene therapy trial conducted at the University of Pennsylvania. Within only hours of receiving his dose, he became ill. Four days later, he died of massive organ failure due to an extreme overreaction of his immune system. (UVM.edu)  In this instance, the researchers should have been more cautious about administering safe dosage; but this doesn’t mean that gene therapy as a whole is a failure. It is done by trial-and-error, like any science or technology.


     There are other issues that are raised by anti-gene therapy groups. According to Chris Wharam, the two main concerns are “playing God” and “the slippery slope.” (Wharam) Of course, with any new scientific advancement comes the concern that we’re stepping outside our natural abilities as humans. Gene therapy has received specifically critical reactions like this because treatments are designed to actually change our genetic code. These concerns are focused mainly on HGGM, declaring that having a hand in shaping our children before they are even born is unnatural, and should not be allowed. One easy counterargument though is that God made the technology available, so this is not going against His will or acting as Him. Another, more viable aspect of the “playing God” argument against gene therapy is that eventually we will be able to change things about our children such as physical appearance, intelligence, or behavior. This is a cause for concern, as the thought of “superhumans” with intelligence like that of supercomputers and the physical qualities of Olympians traipsing around being better than most is unnerving. This sense, in my opinion, could be mostly attributed to humans’ fear of the unknown. If this was to actually happen, and personal enhancement was allowed, there would be a large portion of dissenters in the population. They wouldn’t understand how or why this was allowed, or how it worked, or why people would want to do it. As it became more commonplace, if it were indeed allowed, people would slowly become accustomed to it, and it wouldn’t be a big deal. I would compare this to something like racial equality, perhaps, or maybe the views of homosexuality from the fifties forward. Initially, people would be hostile toward enhancers, or look down on them, but it would become a normal part of live eventually.  I’m sure, however, that as gene therapy becomes increasingly advanced, it will also become increasingly regulated by the NIH and FDA, preventing such occurrences.


     The “slippery slope” argument pertains to the misuse of gene therapy, or using it for things other than its intended purposes. Wharam cites an experiment in which researchers are attempting to genetically alter a chemotherapy patient’s hair follicles to reproduce, so cancer patients won’t have to go bald. He then goes on to say that this therapy could eventually prevent or reverse baldness in non-cancer patients. Wharam then says that, “This example is quite petty, but those against gene therapy cite it as just the beginning in a "slippery slope" towards the development of a superior human.” (Wharam) People could eventually use gene therapy for a wide range of things that have nothing to do with the reasons they were created. Just like the “superhuman” argument, however, I am sure gene therapies will be heavily regulated as their use becomes more commonplace. In addition, the slippery slope is a classical rhetorical fallacy. The University of Texas defines the slippery slope as “an argument that suggests that one thing will lead to another, oftentimes with disastrous results.” Seeing as this is a fallacy, there really is no argument here. I will admit, however, that certain aspects of this argument could potentially surface at some point in the future.


     Another possibility, as I discussed in my essay analyzing James Hughes’ Citizen Cyborg, is the possibility of extreme life extension, or perhaps even immortality. Gene therapy can replace the genes we lose through cell reproduction, thus giving us an infinite lifespan, especially in the case where all disease becomes curable. This is a heavy topic. Is life extension ethical? That is usually the first thought of many when they think about this subject. To answer this question, however, we need to define life extension. One could argue that standard medicine is already contributing to life extension. According to Infoplease.com, average life expectancy in 1900 for an average white male in the United States was about 38 years old. Compare this with the 2000 life expectancy of 74.8. (Infoplease.com) In merely one hundred years, we increased average lifespan in the U.S. by almost four decades. This was all without gene therapy, or enhancement, or any other form of technological augmentation of the human body. I think life extension is ethical. If it can prevent death for years then these years can be put to use to enjoy life. In my opinion, it would be more ethical to extend life than to not.


     This is not to say that I’m completely for extreme life extension. In my opinion, this question should instead be posed as, “Is life extension possible in our society?” I think that lifespans that cross hundreds of years would be socially impossible. After a certain point, people are not able to provide for their families, and need to be taken care of. This is possible today because people only live a couple decades after retirement. However, if extreme life extension happens, there could possibly be hundreds of years that a person would need to be taken care of. The pressure put on the younger generation would be immense, and I don’t think that it would be economically possible to support that many people who don’t work or make money somehow.


     When done carefully and correctly, gene therapy can cure disease, prevent disease, and, since there will no longer be disease, make lives easier and more enjoyable. It can even extend life. Clearly there are risks, but there are risks to any science. In my opinion, scientific advancement should be a welcome and useful thing in any form, because this can lead to more advancement. In order for gene therapy to be a successful medical tool, people need to better understand the science and purpose behind it, and embrace it. It can be a much better world this way.




     In the not-so-distant future, gene therapy will be an everyday medical treatment, available to patients on a whim. This will be a wonderful advancement for many, who have diseases that are untreatable by normal medicine. My hope is that gene therapy does become normal at some point; but, like all readily available medical treatments, it needs to be regulated and monitored to protect against abusers. Gene therapy can have very serious and often permanent effects. As I discussed in my evaluation on gene therapy, there are many uses for gene therapy. Good progress is being made every day on refining and tweaking the techniques and processes involved. It is also on its way to being a safe and easy form of treatment for thousands. There will be problems though, and this is where my plan comes in. It is designed to make a safe and effective environment for gene therapies to function as medical tools. In order to prevent the abuse and harm that could be caused by gene therapy, I’m proposing a three set of laws that would regulate who has access to the medicine, regulate how it is acquired, and see that it can only be used for medical purposes.


     Regulating who has access to gene therapies will be relatively easy. One law would state that gene therapy would only be allowed for those with diseases deemed curable with it. This would of course be diagnosed by a board certified physician. In addition, I propose that any diagnosis of a therapy-treatable disease is reported to a separate agency, whose purpose will be to review diagnoses and, if needed, re-test the patient. This agency’s participation will help prevent medical fraud or malpractice that could happen due to doctor error. I also propose that this agency should be government-based, and get funding from taxes. This would make it easier for hospitals and private practices to get their diagnoses reviewed and approved. It would also speed up the process, because there would be no cost hindering the submission of material. Many of the employees at this agency would be doctors, because there will be a need to correctly interpret and review the diagnoses submitted.


     Gene therapy will definitely not be available over the counter like pain meds or allergy pills. Another law would see that gene therapies will be prescription-only, written by the diagnosing physician. These prescriptions will only be approved after the diagnoses have been sent to the review agency and approved. This is obviously to prevent those who shouldn’t be using the therapies from getting it. Since gene therapies would have to be specifically tailored to each patient depending on disease, it could be disastrous if random users were using gene therapies. This could lead to great harm and death, as I said, so this law would be an attempt to prevent that.


     The third step in my plan will be a law to prevent the commercial sale of gene therapies to people without medical conditions. This will be the most difficult part of my proposal, because there would be a huge potential for all types of therapy to have a wide commercial market. Medical companies would jump at the chance to sell their products to average people, seeing the large profits that could come of it. But, as I stated earlier, there is also a great chance of abuse and harm coming from gene therapy. This would be contradictory to commercial sales, as selling it like any other product would make it extremely easy to get and abuse.  So, as I said before, gene therapies should only be available to people with medical conditions treatable with gene therapy, diagnosed and prescribed by a physician. This could be controlled by a bill passed through Congress. This bill would prevent the sale of gene therapies in any place that does not have a pharmacy. In addition, the medicine will only be passed through the pharmacy, not for sale on the sales floor of a store with a pharmacy, such as Wal-mart or Kroger. Only the pharmacists and pharmacy assistants will handle the products. This may seem a bit extreme, but given my description in my evaluation, it is clear that great harm can come to those who misuse gene therapies. A set of rules such as these would prevent people from getting what they don’t need and have no medical use for.


     In addition, this set of laws should be able to prevent the fear of and probability of superhumans and the slippery slope, as I discussed in my evaluation. Acquiring superhuman qualities would be near impossible under my plan. Getting gene therapy without a medical necessity would be hard. Getting the specific therapy or therapies that one would need to enhance their body would be even harder. The chances that we would see superhumans or the slippery slope in our lifetimes is ridiculously slim, firstly because gene therapy is still in its very early development. Secondly, as I stated earlier in my evaluation, I’m quite sure that some rules would be set in place, hopefully similar to mine, to prevent these scenarios.


     I wish that gene therapy will become a normal medical practice sometime in the near future. It would be a great achievement for our society, but it should be viewed and taken with caution. Though my proposal may seem strict, I only intend it to be in the best interests of society as a whole, not just those with medical conditions. With anything, there is a chance of abuse and misuse. This is why I am making this proposal. As time goes on and gene therapy is more and more common and useful, it will need some rules. I can only hope that these rules won’t hinder its progress.



Works Cited


"Ask CGEP." The University of Vermont. Web. 10 Dec. 2011. <http://www.uvm.edu/~cgep/Education/Expert.html>.


Baruch, S., A. Huang, D. Pritchard, A. Kalfoglou, G. Javitt, R. Borchelt, J. Scott, and K. Hudson. "- Genetics & Public Policy Center || Publications & Resources || Center Reports." - Genetics & Public Policy Center -. Johns Hopkins University, 2010. Web. 10 Dec. 2011. <http://www.dnapolicy.org/pub.reports.php?action=detail>.


Ferrari, Giuliani. "Gene Therapy for Beta-Thalassaemia: Results From First Trial." Thalassaemia International Federation | A Non-profit, Non-governmental Patient-driven Organization, Founded in 1986. Thalassaemia International Federation, 2010. Web. 10 Dec. 2011. <http://www.thalassaemia.org.cy/gene_therapy_for_beta_thalassaemia.html>.


"Life Expectancy by Age, 1850–2004 — Infoplease.com." Infoplease: Encyclopedia, Almanac, Atlas, Biographies, Dictionary, Thesaurus. Free Online Reference, Research & Homework Help. — Infoplease.com. Pearson Education, 2007. Web. 10 Dec. 2011. <http://www.infoplease.com/ipa/A0005140.html>.


Nienhuis, Arthur. "How Does Gene Therapy Work?: Scientific American." Science News, Articles and Information | Scientific American. Scientific American, 13 May 2008. Web. 10 Dec. 2011. <http://www.scientificamerican.com/article.cfm?id=experts-gene-therapy>.


"Rhetorical Fallacies | Undergraduate Writing Center Handouts." Test Page for Apache Installation. University of Texas, July 2006. Web. 11 Dec. 2011. <http://projects.uwc.utexas.edu/handouts/?q=node/30>.


Wharam, Chris. "Human Gene Therapy." NDSU - North Dakota State University. 1999. Web. 10 Dec. 2011. <http://www.ndsu.edu/pubweb/~mcclean/plsc431/students99/wharam.htm>






Comments (0)

You don't have permission to comment on this page.