Genetic engineering is used to change the make-up of genes so that the capabilities of an organism can become beyond what it’s natural potential holds.
It can be applied to all types of organisms including bacteria, plants, animals and even humans. Because the subtopics of genetic engineering are so vast, this essay will focus on exploring gene therapy, a part of genetic engineering that deals with the human organisms (“Gene therapy and genetic engineering”, BBC Bitesize). Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure disease or improve your body’s ability to fight disease.
It holds promise for treating a wide range of diseases, such as cancer, cystic fibrosis, heart disease, and AIDS. Unfortunately gene therapy is not all perfect, this is due to the several possible disadvantages of its procedure. (“what is gene therapy?, U.
S national library of medicine). At this point the medical industry questions whether gene therapy does more good than harm. If genetic therapy has notably helped patients, how so? and if not why? This essay will further explore gene therapy and determine if it has helped change the medical landscape for the good rather than for the worse. To begin gathering the advantages and disadvantages of gene therapy, it is crucial to understand the science behind how it works. Here lays a substantial amount of information that can determine whether there is a high risk of medical complications or not. The science behind the procedure of gene therapy varies depending on each disease however they all have the general need of added, subtracted, reshaped or replaced genes to cure the mutation (“gene therapy”, Gale encyclopedia of medicine). This means that they all need a way to insert their gene. In other words they all need a specific therapeutic gene (A gene that is chosen to be inserted in the cell based on the disease).
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To explain how exactly this carrier does it’s job, I will used an example. Since each disease has a different therapeutic gene, this example will be based off a cancer cell. Imagine the cancer cell is missing a specific gene. To insert this gene scientists have learned that a carrier molecule called a vector can be used to deliver the therapeutic gene to the patient’s target cancer cell. Vectors are viruses and although viruses may not seem like a good thing to inject in someones body, scientists have found a plus side to them. Vectors have the ability to recognize certain cells and carry genetic materials into them. The reason why they do this is because viruses have been naturally programmed to spread their disease.
Just like in a mosquito, vectors travel and are then meant to inject a specific gene in the nucleus of a new cell. Clearly vectors are a natural fit for this position however there are 2 things that make vectors a not exactly perfect therapeutic gene carrier. The first thing that scientists need to change in the vectors is the material that they carry. Once the scientists know what the therapeutic gene is, they will then insert it inside the vector. The second thing changed by scientists is the way the vectors interact with the human immune system. If scientists do not change this second factor the immune system in our body will sense the vector as a threat and will attack it. When the scientists change the appearance of the vector, the immune system will recognize it as an ally allowing the vector to proceed doing its job (“how does gene therapy work?”, U.
S national library of medicine). Gene therapy can turn long term care and treatment to short term permanent fix. Studies support this point, one including a successful trial that worked with patients who had a degenerative blindness called LCA. (Leber congenital amaurosis). In this trial 6 out of 9 patients with the degenerative disease had improved vision after a vector was used to deliver a functional REP1 gene. This case shows the potential of gene therapy and how even at an early stage it is capable of curing diseases (clinical development of therapy: results and lessons from recent successes, sciencedirect ).
Another strong advantage of using gene therapy is that scientists are gradually discovering more and more ways to design vectors. Meaning that with time there may be more and more diseases curable by gene therapy. One of the cases proving this point has been recorded in an article called “methods of clinical development” . The article stated the success of a particular case dealing with a disorder called Severe Combined Immune deficiency (SCID). this disorder was one of the first genetic disorders to be treated successfully with gene therapy, proving that the approach could work. However the first clinical trials ended when the viral vector triggered leukemia (a type of blood cancer) in some patients. Since then, researchers have begun trials with new, safer vectors that are much less likely to cause cancer.
To this date there are several more successfully recorded trials and the few setbacks only made gene therapy a more developed method of curing disease. So not only is gene therapy curing disease at an early stage of experience but it is also learning from mistakes in its trials to further improve its treatment ability (“Gene therapies development: slow progress and promising prospect.”, U.S national library of medicine) Unfortunately there is always a downside to things. As for gene therapy there, there are 4 main risks that patients need to consider. The first risk is an unwanted immune system reaction.
Your body’s immune system may see the newly introduced vectors as intruders and attack them. This may cause inflammation and, in severe cases, organ failure. This occurs because scientists can’t predict how exactly the body will react to the vectors. Even when the vectors or designed to appear harmless, it still may not be enough for some immune systems to recognize it as an ally. The second risk is if the vectors target 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, such as cancer. The third risk is infection caused by the virus. It’s possible that once introduced into the body, the viruses may recover their original ability to cause disease (“challenges in gene therapy”, Learn Genetics). The last risk that patients need to consider before going through with this method is pricing.
This may not have to do with the medical concerns of gene therapy however it is just as important. Gene therapy has proven to be extremely expensive. A gene therapy specified for rare genetic diseases approved in Europe in 2012 costs close to $1 million, and prices are expected to follow suit in the United States. This high pricing limits treatment to those who are not wealthy, and surely there are many people who can’t afford to pay 1 million dollars (“what should gene therapy cost?”, DNA science). Ethics and Economics are 2 things that gene therapy may affect in a negative way. For ethics, the concern is that manipulating factors such as intelligence might be tried, once gene therapy becomes commonplace. Gene therapy may no longer be used for medical necessities and ‘Ordinary’ characteristics, such as shortness or average IQ, might then be considered ‘subnormal’.
The concern for economics lies in the idea that gene therapy might only be available to the rich. As mentioned before, gene therapy is currently available at a very high price. If only the wealthy have access to gene therapy the division between the rich and the poor would become more evident. The people who have enough money may invest in improving characteristics such as intelligence or physical ability while the poor will stay the same.
The challenge for nations experimenting with gene therapy is to come up with workable, fair and ethical guidelines for its use. Through this analysis of gene therapy, it is safe to say that it has several medical, ethical and economical risks in present day. It is dangerous to let everyday patients have access to gene therapy since it is still a developing method of treatment.
There are however high hopes that one day it will be completely accessible, especially after seeing the developing results in the trial involving patients with Severe Combined Immune Deficiency (A trial in which a mistake was made resulting in some patients having leukemia. After the mistake scientists made sure the following experiments were corrected. After doing so there was a rapid positive change in results) that was mentioned earlier. Knowing how quickly and effectively scientists have learned to design vectors in present day, the near future could hold even more advanced cures with gene therapy.
Pricing may also come down in the future since the research money will have been paid off. As long as the ethical and economic side can balance out the same way as the medical side can, gene therapy is potentially the new revolutionary cure to what seems like the incurable.
