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What?s Gene Therapy Ethical? Essay

The question of whether or not it is ethical to change the genes of a child before they are born, to rid them of a family disease or just simply to be able to pick their traits, has been going on for years now. Every year humans as a whole are getting closer and closer to minimizing the major health concerns, so gene therapy can be done safely. It has been done on people in the past, but very few have received an effective treatment. But this doesn’t mean gene therapy is impossible, it just means more research needs to be done.

Gene therapy was first proven possible in humans in 1990 on a four year old girl named Ashanthi, who as suffering from a rare disease. In 1998, author Sharon Begley published her article, “Designer Babies”, where she expresses her feelings on the topic of changing genes. She revisits the idea of altering the genes of unborn babies, which she believes is unethical because of the fact that it would create a massive decrease in diversity. Today, there are many different views on if gene therapy is safe or not.

Researchers in China have found a way to test the gene therapy without it being done on a live human, using a procedure called CRISPR. They use embryos that would otherwise be destroyed to maximize their esearch, without the possibly of harming an unborn baby. In today’s society, research is being done to find new and more effective techniques to do this procedure safely. Throughout this paper, I will be focusing on the ever-changing progress of gene therapy over time. In 1990, the first ever gene therapy trial came about by Dr. William French Anderson.

His four- year-old patient, Ashanthi had a very rare disease, known as severe combined immunodeficiency. Her disease was caused by an absence of an enzyme. This prevented her body from creating white blood cells that were needed to fight off infections. Because of this, Ashanthi got very sick with even the slightest infections, for example the common cold. At this time, there was a drug called antimicrobial that could treat this infection, but not permanently. Before the discovery of gene therapy, there was only two ways to help Ashanti’s disease.

One was getting regular injections of the enzyme that she lacked, and the other was a bone marrow transplant. When she was two years old, Ashanthi started getting the injections and they worked up until she turned four when her health started deteriorating. The doctors thought the next option, a bone marrow transplant would be he best option, because of the fact that bone marrow produces white blood cells, which is what she needed. However, this procedure was not done because of the lack of compatible donors. Ashanthi’s parents stated to panic and needed to find a way to get their daughter healthy.

At this time, permission for gene therapy was being obtained. Gene therapy was already proven possible by scientists on animals and plants, but was never done on a human. The way this procedure worked was that working copies of the enzyme Ashanthi did not have were inserted into her cells with a vector. A vector is used by scientists o input DNA into patients. The vector was a virus that was fixed so it did not cause disease. Within six months of her procedure, she was healthy and her white blood cell count had risen. Ashanthi’s cure was unfortunately short lived.

While she was doing the gene therapy, she was also having enzyme therapy, just in case the gene therapy did not work. In order to see if the gene therapy was really working, she stopped the enzyme therapy, and her symptoms then returned, leading her to stop the gene therapy. Scientists in the 1990’s still had much more to learn about gene therapy before trying it on another patient “Treating the Bubble Babies: Gene Therapy in Use”). Sharon Begley’s article, “Designer Babies” appeared in the Newsweek in 1998. Her article went into depth about altering children’s genes before they are born.

Begley discusses how changing the genes of a fetus could potentially help those with very serious family diseases. At this time though, gene therapy was just found possible and scientists were not yet doing this procedure on a fetus. Begley states, “Since 1990, gene therapy has meant slipping a healthy gene into the cells of one organ of a patient suffering from a genetic disease. Soon, it may mean something uch more momentous: altering a fertilized egg so that genes in all of a person’s cells, including eggs or sperm, also carry a gene that scientists, not parents, bequeathed them” (Begley 116).

Begley mentions in her article that when gene therapy first came about, the government vowed to never alter patient’s eggs or sperm. Dr. W. French Anderson of the University of Southern California stated that, “Within two or three years he would ask approval to use gene therapy on a fetus that has been diagnosed with a deadly inherited disease. The therapy would cure the fetus before it is born” (Begley 116). After Anderson submitted his proposal in September, he received more than 70 pages of comments from the public and scientists, opposing this idea that tampers with the egg and sperm.

Anderson states, “with the exception of a few anecdotal cases, there is no evidence of a gene-therapy protocol that helps” (Begley 118). Just because many people did not support gene therapy during this time, did not mean that the “dream” of gene therapy would stop. This was only the beginning to finding a cure for deadly inherited diseases. Another aspect to gene therapy that has been changing since the beginning is people’s iews on the procedure. Many people believe the idea of “designer babies”, altering the genes of an unborn baby for parent’s desired traits, to be unethical.

The main reason for this is that because it would create massive decrease diversity, because of the fact parents would be choosing their children’s traits. In Bonnie Steinbock’s article, “The art of medicine: Designer babies: choosing our children’s genes”, published in 2008, Steinbock explains how he believes designing your children is morally wrong because children wouldn’t be able to express themselves. Steinbock says, “Perhaps the objection is to he fact that the child’s genes were chosen for him by his parents, thus forcing the child to have certain talents and not others” (Steinbock).

He believed that the child would have no input into what they want to pursue in their life. Part of being a child means trying different things to find the one thing they really desire. If parents genetically engineered their child to be good a musician, that would be the only talent they would have. At this time in history, people, including Steinbock viewed the procedure of designing babies to be very wrong and that it should not be done. The main goal for gene therapy is to help hose with sickly diseases, by altering their genes. Decades of research on gene therapy have led us to designing safer procedures.

Gene therapy so far has been able to treat many certain diseases and be successful for many patients. Several immune deficiencies have been treated with gene therapy, by removing blood stem cells from patients. Another disease that is being treated with gene therapy is hereditary blindness. In a trial of patients with a form of degenerative blindness, gene therapy improved their vision for a few years. Unfortunately, it did not last very long. Another trial came about where 6 out of he 9 patients with degenerative blindness had improved vision immediately after the procedure. In 2016, cancer is the second top leading cause of death.

Gene therapy treatments are currently under development for this terrible deadly disease. In a trial of 59 leukemia patients, a type of blood cancer, the patient’s immune cells were removed and were treated with a virus. After the immune cells were returned to the patients, 26 of them experienced full remission. Parkinson’s disease is another disease being treated with gene therapy. Parkinson’s patients gradually lose cells in their brain that produce opamine. These patients lose ability to control their movements. In a trial of a small group of patients, were treated with new genes that introduced cells in the brain.

The genes gave cells that normally cannot produce dopamine, the ability to do so. After the trial, all of the patients who received the treatment improved muscle control (“Gene Therapy Successes”). Today, gene therapy makes it possible to treat those with deadly inherited diseases, simply by altering their genes. A new and effective form of gene therapy called, CRISPR is being talked about as the “next big thing”. CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats, which is basically the bacterial defense system in the body.

CRISPR can be programmed to target specific genetic codes and edit specific DNA in the body. This enables researchers to permanently modify genes in organisms and living cells, with hopes in the future to be able to modify mutations in human genes to treat genetic diseases (“Project Spotlight CRISPR”). In 1993, Francisco Mojica discovered CRISPR. He worked on it throughout the late 90’s and 2000’s. In 2013, CRISPR set our for genome editing. Feng Zhang, a Broad Institute of MIT and Harvard, was the first o successfully adapt the gene editing in human and mouse cells (“CRISPR Timeline”).

With CRISPR, scientists can create mouse models of human diseases much quicker than before, study single genes much faster, and easily change multiple genes in cells at once. Blake Wiedenheft, a biochemist at Montana State University in Bozeman stated, “I don’t think there’s any example of any field moving this fast” (Pennisi). In 2016, scientists recently got approval from the UK Human Fertilization and Embryology Authority to edit genes in embryos. In 1998, when Sharon Begley published her article, “Designer Babies”, this rocedure was considered a dream. Now, it is a reality.

Dr. Kathy Niakan of the Francis Crick Institute in London, also the American Biologist, leading the study stated, “I was really pleased to hear the outcome. But obviously it does await ethical approval as well” (Chamary). Changing the genes of an embryo is very controversial and raises many ethical concerns, because of the fact that many scientists believe it will lead to designer babies. To study the genes in the embryos, Niakan is using CRISPR. CRISPR allows her to be able to use a group of molecules to cut DNA and insert a different piece of DNA in its lace.

The main reason for this is to be able to fix and change a defective gene in the body, possibly causing disease. CRISPR is used to repair the defective gene so the child would not have a certain type of disease. CRISPR allows scientists to make many different precise changes in DNA. Niakan believes that CRISPR will shed light on how embryos develop and states that, “We can use this new method that’s extremely precise and efficient to ask questions about early development that has profound importance for stem cell biology, and for our understanding of why some embryos fail to thrive” (Chamary).

In today’s society, the new technique CRISPR is known to alter DNA very effectively and accurately, changing the world, as we know it. Gene therapy has come a long way since it was first proven possible in 1990. Today’s technology has allowed scientists and researchers to find new, safe, and effective ways to change the genes of a human, to rid them of a disease. The idea of altering the genes of an embryo was considered a dream in Sharon Begley’s article, “Designer Babies”, published in 1998. Now, the procedure CRISPR is being used to do this procedure safely and effectively.

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