Method for gene therapy

In gene therapy, two different types of methods can be used when it comes to vectors, these are called ex vivo and in vivo. In vivo means inserting the desired gene (s) directly into the cells, whereas ex vivo means inserting the desired gene (s) outside the patient’s body and thus first injecting it back after replacing the missing gene (s).


This image shows an example of gene therapy using an adenovirus vector. The modified vector (s) are inserted into the vector that will transport it (s). In order for the gene to reach the cell nucleus, it binds with a cell membrane as a kind of protective layer and is then packed into a vesicle. A vesicle is a conveyor inside the cell that transports from the outside of the cell to the outside of the cell. The goal of this is that the new gene (s) should be able to provide the information the cells need to create a missing / deficient protein and correct the incorrect information that the body and cells have received.


The first try

The first successful experiment was performed in 1990 on four-year-old girl Ashanti DeSilva who suffered from a certain type of SCID called SCID, the disease is due to a lack or complete absence of an enzyme called adenosine dementia. They then wanted to replace these genes with new ones in order that they would produce more of this necessary enzyme that the immune system needs. This experiment was considered successful as her body produced significantly more of the enzyme, but unfortunately she never fully recovered. Here people became very hopeful and it was regarded as a major advancement in gene therapy, modern treatment methods and development. You could save this girl (even though she was not completely cured) from premature death thanks to even then knowledge that it was precisely the lack of this enzyme that was causing her illness and therefore you could “paste” new genes that produced this important enzyme. A lot of people saw this as something positive, that you could now treat innocent children and that gene therapy actually worked.


Ethical values

Some, on the other hand, saw it as changing a person’s inheritance forever as something negative and too risky, as some people with more conservative ethical values considered this a way of playing god, who really has the right to decide who can live and not, and who really has the right to make such a decision that can affect generation after generation. There is nothing that can be based on facts, but there are different opinions based on different ethical values. There is no right or wrong but everything is only opinions even though some are more sensible than others, some feel that it is something too big for people to make such a big decision and thus such a huge responsibility to influence the lives of many people without being quite sure of the consequences. Continuing with gene therapy trials and continuing development would probably mean learning a lot more about diseases and treating those diseases, while far from all gene therapy trials being successful. The question is, did the researchers and the state really have the right to make decisions that could mean death for patients whose trials do not go as intended, but which can at the same time provide them with enough knowledge to save more lives later. There is no factual basis that many would have to die to be able to cure illnesses later, but that was what many thought, some even thought that gene therapy would not work after a failed attempt at an American eighteen-year-old. The American eighteen-year-old died when he reacted to the virus used as a vector, which made many upset as the vectors are part of the basis of gene therapy and that gene therapy had killed a person before it had saved anyone.


Perhaps the biggest reason why gene therapy is so much debated and limited is that the development itself is not completely clear and we do not know everything yet, for example, you did not know that the American eighteen-year-old would die from gene therapy. Above all, gene therapy does not always work as intended, sometimes things go wrong and people die because of it. There is no guarantee that it will succeed or that the gene will be placed at the exact place you want, it is impossible to predict the exact consequences of changing the genetic mass as it is something that should be observed for a long time. There is also a risk that the gene is not properly inserted into the cell and that an incorrect cell division occurs, which causes cancer. These vectors are not entirely safe, when one would cure boys affected by SCID (2002), the engineered retrovirus activated genes in bone marrow cells which led to uncontrollable cell division and 5 of 20 boys suffered from leukemia. In my opinion, gene therapy comes for a good purpose and as a good idea to cure people, however, I think you are already beginning to see that people are rushing to it some even though it is still not a particularly safe method.


Tfor example, they performed this gene therapy on 20 children (above) without ever seeing the results of the treatment, which led to one of these children dying, one has to study the effects of these treatments for a long time and actually through several generations because one usually can see most mutations as an effect of this treatment when the genetic material is passed on to the next generation. It is a very promising method and the very closest we have come to a cure for cancer, even though it also causes cancer. The problem with such revolutionary methods as this or just the hope that this could cure cancer (since it has already produced good results on head cancer trials) is that these are huge sums of money, a big win for those who manage to develop this method continued. During the trials with head neck cancer, it has yielded much more promising results than just radiation therapy, that gene therapy became a success would therefore mean losses in radiation therapy that would be replaced with newer and more modern ones. Why then do I think it would be a problem that these are huge amounts of money, when it comes to money, and especially money in huge sums, people tend to do a little bit of anything to make this money. In gene therapy, the best thing for them would be to develop gene therapy as much as possible and preferably be the first to do so to that degree, therefore they are usually rushed to these trials which entails a great risk, especially if these mutations in the genetic material would not be noticed and continue to be passed on through generations. We have only studied these trials for a relatively short period since it was not very long since the first trials were performed and I think that one should wait before using gene therapy too much as we do not at all what consequences this will have about say 20 – 50 year. In my opinion, it is very risky to use gene therapy in widespread use when we have no idea how this will affect us in the future, we know that these changes in the genetic mass will be brought about in future generations but if something goes wrong and any mutation happens as a result so they will too.


There is a risk with in vivo, it is that you cannot guarantee that the gene is transported and put on the right cell, the gene has a “mission” to perform and if it ends up completely wrong then it will be defective in DNA and the gene can cause a lot problems such as new diseases and defects. The goal is, of course, that such problems should not arise, but we are not there today, gene therapy is a very promising method but not “finished” today and it is no idea to rush it unnecessarily. For individuals above all, gene therapy in its developed form would be something very positive, when researched more then one could cure a lot more diseases and also prevent a lot of unnecessary suffering by curing and preventing the onset of diseases. For example, it could be that you replace this defective gene or cure this disease so that it is not passed on to the next generation or that you have time to replace the defective gene before doing too much damage, since you can now stop diseases already at this stage. .

For society, it is primarily positive that the population can be cured of previously incurable diseases and this indicates a very developed society. However, the treatments are expensive and as a potential future cure for some cancers, many people and many treatments are involved, one would not have enough money to finance all these treatments and then one would probably need to prioritize them in the greatest need. In Sweden, we in turn have a fair health care system, but it is far from in all the countries you have it, which means that only those with enough money would be able to do this in a large part of the world.


It is a fantastic thing to be able to cure people and to let go of all suffering, it usually seems that the most ethical thing to do is to cure as many people as possible and be able to give everyone a fair chance in life, and of course it is optimal, it is is what a non-profit society looks like. Unfortunately, it doesn’t really look like that in today’s society, we live on an overpopulated planet with lower mortality and significantly higher lifespan than before, which means that there are many more people living on our earth than the earth can handle and what resources there are for . According to natural selection and evolution, they survive best adapted the longest and the strongest genes are passed on, then we are not in need of evolution in the same way anymore, but we are still in need of adaptation as some believe it is the best adapted that will Surviving in the future and defective genes are a sign of poor adaptation. It is then thought that it would be unethical to modify people so that everyone will survive as it will only create bigger problems when the “weakest” will still be “thwarted” however horrible it may seem. If you were to “let” as many people as possible to survive, it would also deplete our resources and nature, then this does not mean that you should just let people die, but you can question how ethical it really is to save so many life as possible and putting such enormous resources and money into getting as many people as possible alive as it creates major problems for people and especially nature, instead of focusing on stopping all the consequences of overpopulation.