Why Is Humanity Still Debating On The Use Of Genetic Modification?

How would you like to better? Usually, people do not get a say in what their genetics say in those three billion letters of DNA that make them who they are. How people grow and develop is shaped by their DNA. We can look at DNA as an instruction manual for how human bodies function. People cannot just decide to adjust their genes in such a way that, in doing so, they gain the ability to increase their strength or speed whenever they wish. However, with new revolutionary technology humanity may finally be able to achieve its goal of enhancing the human body in order to fight diseases and prolong lives.

Many experiments with mice have been done regarding the increase of speed and strength but that is not all genetic modification can do. GM can also be used to treat diseases with a new kind of technology. This technology could help patients with a whole range of genetic conditions. It will not matter if people have caught a disease or if they have inherited it. Their damaged cells could be removed, treated in a lab and then healthy versions can be reinfused in the body. So in order for scientists to begin treating diseases, they need to use gene therapy or gene editing. In my research, I will give examples of several breakthroughs in genetic modification. Like how insects can be genetically modified not to spread diseases like Malaria or the Plague. Also, how GM can also help people who are suffering from different kinds of blood disorder, it is even possible to treat cancer with GM technology. Considering all of the benefits people tend to gain, why is the continued development of genetic modification still being questioned? Are there any threats that we fail to see? That is what I plan to find out.

To begin with, what is genetic modification? GM can also be referred to as gene editing. Gene editing can be pictured as a molecular satnav which scans the DNA in search of any forms of errors. After the error is located, molecular scissors are used to snip through both strands of the DNA which switches off the faulty gene. Finally, gene editing can repair the code by inserting a healthy copy of the gene. There will be more specific details in the examples (Howard et al.).

I will now turn your attention to how genetic modification can help people who are suffering from a blood disorder. A blood disorder is a condition in which the red blood cell, white blood cells, and platelets are not functioning as they should. Red blood cells play the role of transferring oxygen to the body’s organs and tissue. White blood cells help the body fight infections. And platelets are tiny molecules that stick up together and build a clod on bleeding wounds. All three of these cell types form in the bone marrow, which is the soft tissue inside bones. The body needs millions of blood cells produced by the bone marrow in order for it to carry out vital functions. However, sometimes bone marrow cannot work correctly due to certain factors and results in blood cell disorders. Unfortunately, there are inherited blood disorders that keep the blood cells from doing their job, making it harder for the body to function. Since the disorder is inherited, it is caused by a faulty gene within the red blood cells. Researchers are developing a gene therapy that could potentially correct the faulty gene in patients with Beta-thalassemia and Sickle cell disease (Macon, et al.).

Now let us see what the root causes of these two disorders are and how they can be treated through gene therapy. Beta-thalassemia is a wildly spread blood disorder. Approximately forty thousand children are born each year with a severe form of the disorder called beta-thalassemia major. This disorder can cause severe anemia, a condition in which the blood does not have enough red blood cells. Beta-thalassemia is caused by a malfunction in the gene that makes beta-globin, a protein that is used by healthy red blood cells to carry oxygen throughout the body. People living with Beta-thalassemia cannot make enough or even any beta globin. The current treatment is frequent and lifelong blood transfusions that deliver red blood cells from donors to the patient to correct the anemia. However, the transfusions also cause iron to build up in the body, which may cause severe damage to organs, abdominal pain, weakness, fatigue, and joint pain. So, patients who receive blood transfusions must also take additional medicines to remove excess iron. This treatment is called iron chelation therapy (Betа-thаlassemia).

Scientists are researching an investigational gene therapy approach as a potential treatment for Beta-thalassemia major. In the laboratory, a corrected or functional copy of the beta-globin gene is placed into a lentiviral vector. There is a small number of parts from the human immunodeficiency virus or HIV that are in the vector. The HIV parts are used because they are effective at entering cells and delivering the functioning copy of the gene. The vector has been changed so it cannot grow or cause HIV infection. The scientists then take the blood stem cells from the body of the patient. In the lab, the lentiviral vector is used to insert the functioning copy of the beta-globin gene into the DNA of the collected blood stem cells. What follows is that inside the body a chemotherapy medicine is used to remove existing stem and instead place the new ones in their place which contain the functioning copy of the beta-globin gene. When the modified stem cells are returned to the patients’ body, they will be able to establish a home and multiply. The goal is for the modified blood stem cells to become a permanent source of new blood cells with a functioning copy of the beta-globin gene. This investigational gene therapy approach may potentially help patients produce healthy red blood cells and decrease or eliminate the need for blood transfusions (Nienhuis, Derek).

We will now move on to Sickle cell disease. This disease is an inherited blood disorder. It affects millions of people worldwide and is the most common inherited blood disorder in the United States, affecting about one hundred thousand Americans. Screening for Sickle cell disease is typically done at birth. People with Sickle cell disease have abnormal hemoglobin. Red blood cells are where hemoglobin can be located. Hemoglobin is a protein which delivers oxygen to all cells of the body. The irregular amounts of hemoglobin cause the red blood cells to become rigid and take on a sickle or crescent shape. The sickled red blood cells get stuck in the body’s small blood vessels. As a result, cells and organs cannot get the oxygen they need which can lead to organ damage and failure. It can also cause episodes of pain known as crises. The most common treatments for Sickle cell disease include antibiotics, pain management, IV fluids, blood transfusions, Hydroxyurea, and surgery. Even though transfusion helps by providing red blood cells, we already estimated that there is also an increase in iron which means that the patients must take extra medicine (Chekroun et al.).

The process by which scientists treat Sickle cell disease is similar to how they treat Beta-thalassemia. In this case, a hemoglobin functioning copy is placed into a lentiviral vector. The scientists use the patients’ blood stem cells which are collected from a bone marrow harvest procedure. The blood stem cells are separated from the bone marrow, and then the functioning copy of the hemoglobin gene is inserted into the DNA of the collected blood stem cells. Inside the body, like in Beta-thalassemia, chemotherapy will be used to get rid of old stem cells and replace them with the new ones. The modified new stem cells will go back into the patients’ body where they are expected to grow and create new cells that contain normal hemoglobin. The idea is for them to become red blood cells that do not sickle in order to prevent organ damage and other complications (Steinberg, Paola).

Now that we have seen how genetic modification will help people with blood disorders, we will move on to cancer. This is a more recent discovery. As we all know, cancer is no one’s favorite topic: the prospect of abnormal cells dividing uncontrollably in the human body and destroying healthy tissue. However, recently a new drug has been created that has the ability to turn one virus against another. Viruses are tiny, so it is tough for them to be spotted by the immune system. Moreover, because viruses are nothing, but genetic material wrapped in a protein coat, they cannot be expected to survive without a host. By attaching themselves to other cells, viruses can only then multiply. The cells that the viruses have latched on to can then be reprogrammed to create new viruses until the cells themselves die. Cancerous cells can be accidentally created because of this process. However, it is the viruses’ ability to infect and kill cells that make them an ally in the fight against cancer, because GM can alter the way viruses behave. There are scientists who are testing out the new drug for skin cancer called T-VEC. T-VEC is herpes simplex virus genetically modified to only replicate in unhealthy cancer cells, destroying tumors while leaving healthy tumors intact. Scientists inject T-VEC right into the spot where the tumor is located. Even if the tumor is at an advanced stage, the drug is still injected in the same place. Once the cancer cells blow up, a secondary auto-immune response is triggered. T-VEC also has a gene that possesses a type of protein called GM-CSF or granulocyte-macrophage-colony-stimulating factor, that recruits immune-boosting cells to the tumor. Together, killing the cancerous cells and bringing more tumor-fighting cells speed up the drug’s cancer-fighting effect while also stimulating the immune system, waking up the body to attack the cancerous cells. According to the scientists’ research, the drug even has the ability to destroy cancerous cells that have somehow moved away from their previous location in the body, stopping the spread of cancer (Kaufman, et al.).

So far, we have seen what kind of effective gene therapy will have on two kinds of blood disorders and cancer. During my research, I was not able to locate any drawbacks in these examples. However, my final example will be a different story. Now we shall see what effect GM will have when it is used to modify the environment so that the environment itself will need to adapt to humans.

What if people could use genetic engineering to finally eliminate a very dangerous predator for humanity? Said predator is by far the deadliest creature on the planet, the mighty mosquito. The mosquito plays host to Malaria, one of the cruelest parasites on Earth and possibly the biggest killer in human history. Not long ago Malaria was responsible for causing the death of over half a million innocent people. Microorganisms are responsible for the cause of the Malaria disease: Plasmodia, very strange microorganisms that consist of just a single cell. Plasmodia are parasites that rely entirely on mosquitos. Malaria always starts with a mosquito bite. In the mosquito’s salivary glands, thousands of sporozoites wait until the insect penetrates a person’s skin and immediately after invading said person they head for the liver, where they quietly enter big cells and hide from the immune system. For up to a month the sporozoites stay hidden in the cells, consuming them while they are still alive and then changing into their next form: small drop-like merozoites. These merozoites multiply and generate more and more of themselves until the cell cannot take anymore and is eventually destroyed. After the parasites are finished with the cells, they head to the bloodstream in search of their next victim, Red bool cells. However, in order to stay unnoticed, the parasites need a disguise. Said parasites wrap themselves inside the membrane of the cells they killed. Simply put, they use the skin of the cells as camouflage. The parasites now attack the red blood cells by using the same tactic they used to destroy the previous cells. The parasites again multiply inside the red blood cells until they kill them as well. This cycle continues. Lots of toxic waste material can be spread because of the dead cells, which can cause flu-like symptoms because they cause the immune system to respond. The person infected will experience high fever, sweats and chills, convulsions, headaches and there can even be vomiting and diarrhea. If Malaria reaches the blood-brain barrier the infected person can go into a coma, experience neurological damage or in the worst case scenario die. Finally, the parasites are ready to evacuate the human body. When another mosquito bites the same infected person, the parasites get a ride, and the cycle can start over (Phillips et al.).

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The mosquito is very efficient at spreading human diseases. Mosquitos have been around for over two hundred million years, there are trillions of them, and a single one can lay up to three hundred eggs at a time. Mosquitos are practically impossible to eradicate, and the perfect parasite taxi. However, GM technology could help to eradicate malaria forever. For said technology, to work people need to engineer a whole new animal population. Thankfully this is not a hypothetical problem. Modified mosquitos already exist in labs. By using genetic engineering, scientists were able to create mosquitoes with built-in immunity against the malaria parasites. Humans were able to do this by adding a new antibody gene, that targets plasmodium explicitly. These modified mosquitoes will never spread Malaria. And this is only the first step. Malaria is only the beginning. This cure could only be the start of a better future for humanity. A future filled with no fear of diseases. So, are scientists still debating whether to use this technology or not (Boëte)?

Even though people tend to gain a lot by genetically modifying mosquitoes, there is still something missing. In order for mosquitos to stop spreading Malaria, simply changing genetic information is not enough. Half the offspring would only inherit the edits because most genes have two versions inside the genome as a fail-safe. Because of said fail-safe only half of the offspring will have the anti-malaria gene. In a population of billions of mosquitoes, the ones carrying the engineered gene would hardly make a difference. There would not be any point of using this technology if the results were to end up like this.

Moreover, there are other risks as well. Since humans still do not know enough about gene editing, there is a possibility of them making a mistake. Humans have never attempted to change the genetic code of an organism on such a vast scale. Sadly once the deed has been done, going back is impossible. So, genetic engineering has to be done right, because there could be unwanted consequences if scientists set out to edit nature. Not one person would want a giant mutant insect flying around after all.

However, a genetic engineering method called the gene drive has the potential to solve the problem of the two gene versions in the genome of the mosquito. The gene drives force the old gene to be the less dominant one in future generations so that the new gene could take over. Thanks to this technology nearly all of the engineered mosquito’s offspring will possess immunity against malaria parasites. In order for the malaria blocking gene to be spread in the wild, the new immune mosquitoes need to be released so they can mate with regular mosquitoes. Once that is done Plasmodium will no longer be able to use the mosquitoes as hosts. Scientists hope that the change will be so fast that Plasmodium will not be able to adapt to it quickly enough. Malaria could virtually disappear. Some scientists argue that they should use this technology sooner rather than later. The mosquitoes themselves would probably only profit from it. After all these insects do not have anything to gain by carrying parasites (Rasgon).

Regarding the claim about the additional risks to genetic modification, there is a way to bypass said risks. It is correct that there are real risks, but in the specific case of Malaria though, the risks can be acceptable since the genetic modification does not make a significant change in the overall genome. Only a particular part is changed. Simply put, the worst-case scenario would be that the overall treatment will not have any effect on the organism. However, this is a scenario that only concerns Malaria. Scientists still do not have enough information to know how it will affect other forms of the disease.

We now see that there are indeed some valid concerns regarding the development of genetic modification. If the treatments are not handled with perfect care, there may be unwanted consequences for humanity in the form of unwanted mutations or possibly death.

In addition to all of this information, I will also give some information about a recent new revolutionary technology. I will be mainly focusing on the reasons why humanity is not ready to use this technology. Since we have already discussed a possible positive future, I will now show you that there are darker versions too.

The problem for scientists was that, even though they knew that they were able to control what was being edited in the DNA, they still did not know where in the genome that new DNA was being inserted. What scientists needed was a tool that was so precise that it could make a single change I three billion DNA bases, and cheap enough that everyone could use. It is called ‘Clustered Regularly Interspaced Short Palindromic Repeats’ or CRISPR for short. This technology has the power to reshape humanity, combining unnatural selection with non-random mutations. CRISPR is able to cut any genetic sequence down to a single DNA needle in a three billion letter haystack, and then scientists are able to edit the DNA however they please. Genes can be switched on or off. Like for example, a gene that produces too much protein can be turned off. Infections like HIV can be cut out. Humanities’ own immune system can even be reprogrammed to hunt down cancer cells. Mutations in single genes cause thousands of human diseases, and each could be reversed with CRISPR (Hajian et al.).

CRISPR technology was also used in order for scientists to develop the mosquitoes to be immune to the Malaria parasite. Instead of attacking isolated groups of insects, CRISPR can simply change the types that transmit diseases. For the first time, scientists have a tool with the ability to make incredible changes to entire species. So, why are people not using this new technology yet?

With all the hubbub about CRISPER in the news, it may feel like having a disease-free life is just around the corner. CRISPR is a promising technology for treating inherited disorders, cancer, and other diseases. However, this seemingly miraculous gene editing technology may not actually be as straightforward or as safe as people thought. In fact, in the US the FDA placed a ‘clinical hold’ on the first proposed CRISPR gene editing human trail. A couple of released studies suggest that people need to be more cautious when experimenting with genetic modification. Two recent studies explained that when CRISPR cuts DNA, that can cause damage that can accidentally kill the cell, or stop it from developing. It is not certain that CRISPR modifications will be able to kill the defective cells of the gene called p53. What p53 does is that it prevents the onset of cancer by regulating a cell’s life cycle. So, CRISPER may be inadvertently raising the risk of cancer in the patient by leaving more of the defective cells alive than the healthy ones, which is the opposite of the goal. There are also other studies that raise concerns. Up until now, CRISPR’s cutting function has been accurate in the specific area of interest- the spot in the DNA that is supposed to be cut. However, that is because researchers were only looking for mutations caused by CRISPR in the immediate vicinity of the cut. New research reveals that in about twenty percent of cells, CRISPR results in much larger deletions than it was thought- up to more than a hundred base pairs. Researchers were not able to detect any mutations, but that was because the entire region was missing. In CRISPR treatments that would target billions of cells inside the human body, this could also lead to a risk of cancer. CRISPR permanently alters a persons’ genome, so scientist need to make sure that they will get it right before real tryouts on humans are made. However, people are not at the end of the rope just yet. Scientists need to take a step back from the hype and carefully realize what is really going to be safe as humanity moves forward (Haapaniemi et al.).

CRISPR is indeed powerful, but it is not infallible yet. Wrong edits happen as well as unknown errors that occur anywhere in the DNA and might go unnoticed. Humanity just does not know enough about the complex interplay of genes to avoid unpredictable consequences. Working on accuracy and monitoring methods is a significant concern that needs to be taken into account before any human trials can be done.

Technology as powerful as genetic modification needs to be handled with much care, but at some point, people need to ask themselves: Is it unethical not to use this technology when every day one thousand children die? By not using GM, children with inherited blood disorders are denied the chance they have at living. However, at the same time, by not using GM, more lives may be saved since scientists still do not entirely understand about the full extent of this technology. It is understandable, even though scientists possess the tools to change the world, why there is still much debate over the use of genetic modification.