Winner of the Spring 2019 StMU History Media Award for

Best Explanatory Article

Best Article in the Category of “Science & Technology”

You probably have a loved one that has suffered or is suffering from a disease that is painful and detrimental to human health, like Diabetes, Huntington’s, Cysitc Fibrosis, or Sickle Cell Anemia. Imagine if these diseases could be removed from our genome entirely. This is what scientists have been trying to do for years and they have been getting closer and closer. The problem is, once everything gets figured out and can be put into practical use, there are still a number of ethical concerns about applying the method to humans.¹ This method I am referring to is the clustered regularly interspaced short palindromic repeats or CRISPR method. Its discovery has led to a huge potential in editing human genes because of its accuracy, efficiency, price, and its use and accessibility. These factors are what make it such a useful tool for research in editing the genes of all types of organisms, including humans.² What has also helped make the CRISPR method much easier to use is the fact that we have already sequenced the entire human genome before. This, of course, cost the US government around four million dollars and fifteen years to complete. Since then, it now costs as little as one thousand dollars for a genetic study.³ Other methods of course have been found, like zinc-finger nucleases (ZFNs) and transcription activator-like effector nucleases (TALENs), but they are more costly in both the price and the time than the CRISPR method offers and demonstrates. Because this method has become so accessible and useful, more debate and attention are being directed toward the use of it for benefit of human cells.⁴ With the CRISPR method, rather than just adding a copy of a healthy gene into a human, we now have the ability to cut, replace, and patch a defective sequence of DNA. The CRISPR method involves the use of a guide RNA molecule that targets a sequence usually with Cas9, a DNA endonuclease enzyme, that will cut the portion of the DNA sequence that contains the mutation and will put a custom patch in place of it.

The development of this method is credited to Jennifer Doudna and Emmanuelle Charpentier. They earned a multimillion-dollar prize through their research of this technique. However, the first patent for the method was given to Feng Zhang because he is the one who showed the implementation of the method being used to edit the genes of human’s cells.⁵ In one of his papers titled, “Multiplex Genome Engineering Using CRISPR/Cas Systems,” the CRISPR/Cas system is explained in much further detail. They also discuss how this technology has already been tested in mouse and human cells to make way for efficient gene editing. An independent study has established that the CRISPR system has shown a high quality of efficiency when used on human cell lines.⁶

Gene editing in germ line cells, which are cells that develop onto the egg of sperm, is what is extremely important in this research because these are the cells that can be edited so that children will not contain a certain disease or diseases. This is known as germ line gene therapy. By using germ line gene therapy, this will be able to eliminate the use of just somatic gene therapy every time the mutation arises because the mutation will not only be eliminated from the one person but from the generations to come. Somatic gene therapy is the transfer of genes of the somatic cells, like bone marrow cells, which would mean that the DNA does not enter the egg or the sperm and therefore, does not get passed down to the offspring. Scientists have stated that the advancement of gene therapy is necessary because with it, we are able to prevent genetic disorders that we normally would not have even been able to previously identify with the modern tests we have in place now.⁷

A debatable problem arises when we consider whether the modifications on the sperm or egg cell are indeed germ line or somatic. Science historian Nathaniel Comfort writes, germ line modifications “are not used to treat disease in an individual, but to prevent it (or lower the risk) in future individuals.”⁸ He is writing this to give one of the reasons why germ line mutations are said to be ethically distinct. However, it becomes hard to classify the type of modification it is when we look at who or what counts as an individual and what the intentions are of the person(s) issuing the therapy. If an early embryo were to be edited, this would be classified as somatic gene therapy because it is not part of the sperm or egg cells; however, if an adults’ egg or sperm producing cells were to be edited, then this would be classified as germ line therapy, since it will prevent disease in the offspring. But wouldn’t the somatic gene therapy do the exact same thing? Editing the genes of an early embryo would not only prevent that person from getting a disease but would also affect future offspring of that individual. The ethical aspect of which type of modification it is becomes indistinguishable because they interfere with each other.⁹ With this, we will also eliminate abortions. Nowadays, mothers are able to choose whether they want to carry the baby to term if they are told that the baby is going to have a certain disease. With the gene editing method, it will be able to be prevented before this and therefore, there will no longer be a need to abort the baby due to medical issues within the child. The disease could be edited out of the child’s genome before birth. A child having a certain disease will no longer be one of the reasons for abortion. The CRISPR method could ultimately decrease the diseases being carried by humans and passed from generation to generation. If we were to refuse to put this method to use, would we not be violating good patient care? We know that this type of technology is out there, and it could be extremely beneficial to us. So we should take advantage of it.¹⁰

According to Gregory Stock, the most common argument made against the use of gene editing technologies is that it is morally wrong, because gene editing is crossing religious boundaries. However, humans are constantly using technologies like penicillin, birth control, cars, and telephones. These things are all used to help control nature, so why is gene editing set apart? God is the source of where we get anything in science, so how is it crossing moral boundaries to use these things that we were given together to benefit the human race by ridding us of things that harm us?¹¹ A consultant to the Presidential Commission on Cloning states, “human beings are the most important part of God’s created universe . . . God has entrusted this world to humankind’s hands, and the destiny of this world has always been our responsibility and our challenge. Whether or not we live up to that challenge is our calling and essential mission.”¹² Let’s say that there is a person just born with cystic fibrosis, and he could have had the disease removed before birth, but since people decided that it would be morally wrong to tamper with the genetic material of another person, that person was left alone. Then twenty-five years later you meet the person and tell her you are sorry, that her disease could have been prevented, but we wanted to respect her right to her own genetic material not being altered and fiddled with. Is this not morally wrong? Is there not a moral golden rule that says, treat the person as you would want to be treated? If you could go back and edit out the disease either you, your children, your grandparents, or cousins have or had, would you not? What normal human being wants to see a person suffer their whole life due to a disease? Absolutely nobody.¹³

One of the arguments against gene editing is that the effects that it can cause would be irreversible. It is not possible for scientists to know all the risks of editing our genes and since many genes code for several proteins, this is where it can be really dangerous. Scientists will never be able to guarantee one-hundred percent safety because of this issue. There is also the issue that the effects will be passed on to further generations, and of course, the thought of this scares people away even more. What if they are getting rid of the disease in their daughter and she now is a carrier for another type of mutation, but it only gets expressed in males. Her son will then inherit the illness or disease and it would have been the grandparent’s fault because they were the ones that initially decided to edit the mother’s genes. Maybe, in the moment we do not know enough about the safety and precautions of this new technological method, but decades from now it may not be this way. We may have a certain method that ensures no harmful effects and no passing down of any harmful effects either. With the level that technology and science that is progressing now, this may be sooner than we think. Although people will disagree with the application of this method in humans, somehow when the situation becomes personal people tend to change their minds and would then suddenly be on the side of gene editing for their children. No matter how much theologians, ethicists, and scientists debate about the advances in editing technology, the question of whether genetic enhancement is right or wrong will never be completely figured out. People always have different opinions and in the modern world, we rely mostly on the tolerance of people to make our decisions, and that will most likely be the way we either accept of reject these new methods.¹⁴

Of course, there are concerns with the implementation of this new technology, as there is with any advancement in our technological world today. The amount of control that this method could give humans is remarkable when you put some thought into it. We can eventually control traits and properties of humans with this technology. What is to stop people from choosing good genes for their children? What if people decide to edit genes into the human genome, but it is also working towards the prevention of a disease? That would be changing the human genome. This is where limits will need to be decided upon and set. One limit set would be a line where we consider what gene editing is towards the advancement of the human and what is not. Once we surpass the main arguments, what else needs to be considered is “whether we have the wisdom, ethical commitment, and public policies necessary to apply these technologies in a manner that is equitable, just, and respectful of human dignity.”¹⁵

Overall, only when germ line editing increases our physical and mental abilities when it is used will there be a problem. The science has proven right, that it can be successful in editing the harmful or deleterious mutation out of the genome and replace it with the normal one. So we know germ line editing is possible; however, what we do not know is how to deal with it because with it comes its personal, social, and political consequences. One fear that people have as well is the amount of control it will give to the government with the advances of technology. Critics will ask, “what if the government will initiate genetic programs to shape the genetics of citizens.”¹⁶ Critics also worry that parents will make bad decisions based on advertisements for their children, because they think it is the right thing to do. However, with any new advanced technology their are always going to be worries. Things can always go wrong and their is no definite way to make sure they don’t. We are all humans and at one or several points in our lives, we make mistakes. Just take a look at computers and telephones. They have brought us some great advantages but there is also the other side. People can do a lot of tracking, stalking, hacking, and a lot more dangerous things through the internet. No matter how many things are put into place to prevent people from doing the wrong thing, they end up doing it. It is inevitable. What we need to do is weigh out the benefits and the risks of this genetic engineering advancement and make sure that the benefits do out number the risks.¹⁷

Gregory Stock, biophysicist, best-selling author, biotech entrepreneur, and the former director of the Program on Medicine, Technology and Society at UCLA’s School of Medicine makes a great point: “But if advanced germinal technologies will eventually arrive anyway—and even critics generally agree that they will—we must ask which is safer: a path that drives the technologies underground and out of public view, or one that explores them openly; a path that pushes their development into the hands of rogue scientists and states, or one that keeps them in the scientific mainstream; a path with surreptitious funding inspired by visions of black-market profits, or one with aboveboard financing and open-market incentives and constraints.”¹⁸ When we deeply consider all these options, there is one that sticks out and seems like the right way to go. We need to keep the germ line editing technology out in the open because this lowers the risk of people using it for wrong or using it improperly behind the scenes rather than having it done in the open correctly. This will force us to look into any problems that are happening when we first implement this technology. People will also argue that if the technology fails on one child, this will be one too many, but if we thought this way about everything, we would never advance anywhere. Planes or vaccinations would not be existent if we were to do this. What we should be fearing is the fatal diseases that once killed millions, like smallpox and the bubonic plague. Technological advances helped rid us of some diseases like these entirely. The only way we are ever going to verify safety with this new genetic technology, is through the implementation of it.¹⁹