Genetic therapy is the insertion of genes into an individual’s body in order to treat a disease or change a feature of a person. People can now choose to have a child treated before the child is born in order to change what attributes the child has. It is not one hundred percent effective but almost always can a doctor give your child blue eyes if the patient wants a child with blue eyes. Some believe this to be wrong, and that the selection process should be left to God and only God. There are many out there who believe that this development is one of the greatest man will discover because scientist can now change a child’s genetic makeup before the child is born. This can save a child’s life, but this can also cause unseen mutations.
Dr. Martin Cline conducted the first research into gene therapy in 1980. Dr. Cline combined the bone marrow of two subjects. Dr. Cline's study called Splicing Life was not approved by any government. Dr. Cline did not dispose of his experiments to the International review boards as he was supposed to and in turn was forced to resign his department chairmanship at UCLA. He also lost some of his grants that he was awarded for his research. Due to Dr. Cline's experiments which were not authorized, the President's Commission for the Study of Ethical Problems in Medicine and Biomedical and Behavioral Research began a study called Splicing Life. This was the study of the ethical issues involving splicing Deoxy Ribonucleic Acid (DNA).(Splicing Life)
The first approved genetic therapy encounter was on September 14th, 1990. A group of scientist worked to find a cure for a child named Ashanti DeSilva whom had a rare disease called Severe Combined Immune Deficiency (SCID).(Georgetown Bioethics) This disease makes any passing germ fatal to Desilva because she has no immune system to fight off the germs. The researchers then took some of Desilva’s white blood cells, allowed them to grow in a lab and then inserted the missing gene into them. This allowed these white blood cells to successfully fight off the sicknesses that Desilva was experiencing. This cure was not permanent and Desilva had to return back to the scientist almost every other month for the treatments. (Thompson, Larry)
The positives of Gene therapy are tales of those such as the Desilva case. Gene therapy is being used in many ways today, such as inducing genes into a person’s body that will speed up the healing process of scarred tissue. Supplying the patients with genes that will help fight, or help revert cancer cells back to normal cells. They can also help deliver vaccinations throughout the body. The most common use of gene therapy is to replace genes that are missing in the person’s body. The therapy is also being used to help remove mutated genes from a patients system and replace them with normal genes.
Gene therapy research is on the rise all over the world. Researchers are searching for new genes in which they can use in order to make the world a healthier place. The genes in which are being experimented with are Cystic fibrosis transmembrane conductance regulator or CFTR which regulates chloride in the body.(Uniprot.org) CFTR is used to help treat cystic fibrosis .According to Dr. Frederick Hecht they are also using genes to treat Hemophilia, a disease which makes blood clotting harder for an individual. (Medicinenet.com)
Genetic therapy is being rejected by many because those who reject it are saying that scientist is playing God. In a sense they are correct. The fact that ninety percent of the time that researchers decide to changes the genetic makeup of an individual. The genetic makeup of an individual is determined by heredity. Half of the genetic makeup is made from the father of the child, the other half the mother. If we introduce a third party into the embryo to change the makeup then it is introducing to a possible mutation into the baby. The issues following gene therapy are that there is not sufficient enough data to say what can happen to a patient long term. The patient may have severe side effects that lay dormant for decades.
Gene therapy is only being used on patients when no other cure is available to the patient. The patients that undergo gene therapy are those who are suffering from various forms of cancer, hemophilia, and cystic fibrosis is some examples. The treatments are risky and are not one hundred percent effective. There are a few ways to get the necessary genes to the correct place in the human body. One way is to attach the gene like a virus to a protein. Once the virus is introduced into the body the gene is meant to spread to either replace or fix the damaged or missing gene. It can also be introduced as a pill to help it spread throughout the body. Both of these methods are risky and cannot be guaranteed.
The focus of Gene Therapy now is the cure of diseases that are caused by genetic mutations or a genetic predisposition. Once researchers are able to identify and cure the diseases now with little to no side effects the researchers will shift their focus to the prevention of such diseases.
The Food and Drug administration has not yet released any gene therapy products into the world for consumer purchase. All genetic therapy products are still undergoing testing. In 1999 the gene therapy research experienced a setback caused by the death of a patient. 18 Year old Jesse Gelsinger died four days after starting his experimental genetic therapy treatments. He died from multiple organ failure. It is believed that the cause of his death is due to the gene that was introduced to his system. His system rejected the gene and in an attempt to fight the gene his body shut down. Since this incident there have been other more successful treatments as well as new ways to introduce the genes into the system. (Lehrman, Sally 1999)
In London doctors treat young patients who have the disease X-SCID, or X-linked severe combined immune deficiency.(Arthur, Charles 2002)These patients receive treatments of a harmless virus that moves bone marrow throughout the system in order to jump start the patient's immune system. In France 12 children have undergone successful treatment. On September 16th 2002 a child was found to have leukemia after having treatments of the gene to help him with his X-SCID. "It's an ethical dilemma," said Professor Norman Nevin "To deny gene therapy to them would be unethical, provided the parents are cognisant of the associated risks." Now doctors know what caused this rare case and are working to prevent the issue from ever arising again. (independent.co.uk)
Genetic therapy is a very risky business. There are about thirty five thousand genes in the human genome and to find a gene that is malfunctioning of not there at all is very hard to do. For the most part doctors do not know what every gene does so they are mostly considered to be in a gene graveyard. The genes placed in the system by doctors are placed into a lottery. These genes may hit the designated area, or they may miss completely and the doctors may have to try again. If the gene hits the designated area then the genetic transplant will be finished successfully. If the gene misses there are many outcomes. The first outcome is that doctors just have to give it another chance and try again. The second is that the gene placed may turn on the wrong gene and cause diseases such as leukemia. The last option is the worst which is that the body may reject the entire procedure and will fight the gene placed in the system and end in death. Death so far has been a rare occurrence but two patients have died due to gene therapy. Jesse Gelsinger whom was mentioned and Jolee Mohr. Ms. Mohr was being treated for rheumatoid arthritis died after participating in a gene therapy trial. The National Institute of Health or NIH has one hundred thirty nine active cases of gene therapy of which all but ten are to cure a terminal illness."Until things are worked out, it seems prudent to limit yourself to serious disorders," says Jefferey Chamberlain , a University of Washington gene therapist who specializes in muscular dystrophy.(Wired.com)
Some specialist such as Jefferey Chamberlain can agree with those people who believe gene therapy should only be used in extreme situations. As he has stated it should only be used for serious disorders. Some of the less serious disorders gene therapy is being used for are erectile dysfunction, cholera and intermittent claudication. For intermittent claudication gene therapy may be used but it is very risky. It is very risky to test the genes on healthy patients let alone patients who actually have the disease. Intermittent claudication is a complication of arterial disease that can cause severe, potentially disabling limb pain. Even though the patient is in extreme pain the therapy procedure could cause a fatality if all safety measures are not taken. Even with all safety measures take death can occur. (Wired.com)
With all the risk involved with genetic therapy one might ask why even undergo the procedure? Most procedures done using this method are usually experimental and are being tested to see if the procedure can cure the disease. Most of the patients who undergo these treatments are born with these diseases. (Wolf, Jon) Why are they so desperate to cure themselves? Some of the diseases which are those such as some forms of cancer and SCID have no known permanent cure. Therefore genetic therapy is giving the patients who are suffering these illnesses hope. They hope that by replacing some of their more defective genes they can battle against these diseases and make a full recovery and live their lives to the fullest. Children are most affected by diseases that gene therapy is used for. Gene therapy is used to treat children mostly because usually the child dies before the child can grow to be an adult. Gene therapy is very risky on any person and should not be taken lightly.
Children are the main focus for gene therapy. They are because with all of the genetic mutations that occur from generation to generation the children need to stay healthy. Children who are born with diseases such as cystic fibrosis, aids, and cancer are the main target for gene therapy. There are some parents who use gene therapy in order to choose what they want their children to look like. This has been said to be immoral by large groups of people. There is approximately a fifty fifty split in people who are for and against the use of gene therapy. (NDSU.edu)
The pros to gene therapy are it can treat patients that have no other options for treatments. There are some cancers such as lung cancer and breast cancer. These cancers are treatable but are not one hundred percent curable. With gene therapy there may be a cure to these so called incurable diseases. Gene therapy can sometimes offer a true cure and not just a temporary treatment. In some cases diseases are cured in others such as Desilva's case they are temporarily fixed.(Juengst, Eric T) There are also many cons to using gene therapy.
The cons to using gene therapy is it is a fairly new technique. There has not been enough conclusive data that will allow us to say it is completely safe for people to undergo the treatments with no side effects. There is also the risk of death. Death so far has been uncommon, but it has happened. There is also the risk of creating another medical condition such as leukemia. These are all things that can happen while undergoing gene therapy, but are they worth the risk?(Leenen, H.J.J.)
Gene therapy is a relatively new science. The side effects for gene therapy are unknown and yet people undergo these experimental procedures in hope for a better life. For some people the life is better and for others it can actually make life worse. Gene therapy is like playing the lottery, a patient will never know what they will get once they undergo the procedures. Children are the main focus of these procedures. The problem with these procedures is not that we cannot find a cure for the diseases in which we are making an attempt to treat, but the fact that we do not know a vast majority of the human genome. There is thirty five thousand genes in the human genome and scientist cannot identify most of their uses. If scientist can learn what the use of every gene in the human genome is then genetic therapy would become more of an absolute cure as opposed to a cure by chance. Its true that a scientist can make an attempt to combine one humans genetics with another to create the so called perfect being, but that would be playing God. The reason a lot of people are against genetic therapy is because they believe the scientist are making an attempt to play God. Some are, by giving parents the options of having their children be six foot tall and athletic by genetics, or choosing them to have blond hair and blue eyes even though neither parents has those features in their blood lines. The problem with this is because this is a new science side effects are unpredictable and could cause major health problems in the end. Mutations are a big problem with these genetically altered persons. Nobody wants a person to be genetically altered and have a mutation which could bring about another incurable disease.
Works Cited
"Analysis: Doctors Struggle with the Ethical Dilemma of Gene Therapy - Science, News - The Independent." The Independent | News | UK and Worldwide News | Newspaper. Web. 15 Dec. 2010. <http://www.independent.co.uk/news/science/analysis-doctors-struggle-with-the-ethical-dilemma-of-gene-therapy-607786.html>.
"Cystic Fibrosis Transmembrane Conductance Regulator - Homo Sapiens (Human)." UniProt. Web. 15 Dec. 2010. <http://www.uniprot.org/uniprot/P13569>.
"The Ethics of Gene Therapy." NDSU - North Dakota State University. Web. 15 Dec. 2010. <http://www.ndsu.edu/pubweb/~mcclean/plsc431/students/bergeson.htm>.
"Human Gene Therapy." The Bioethics Research Library at Georgetown University. Web. 15 Dec. 2010. <http://bioethics.georgetown.edu/publications/scopenotes/sn24.htm>.
Juengst, Eric T. Human Germ-line Engineering. Dordrecht, the Netherlands: D. Reidel, 1991. Print.
Keim, By Brandon. "Gene-Therapy Deaths Raise Ethics Issues." Wired.com. Web. 15 Dec. 2010. <http://www.wired.com/medtech/genetics/news/2007/08/gene_therapy>.
Leenen, H.J.J. Genetic Manipulation with Human Beings. Web. <http://bioethics.georgetown.edu/publications/scopenotes/sn24.htm>.
"Splicing Life: A Report on the Social and Ethical Issues of Genetic Engineering with Human Beings." Splicing Life: A Report on the Social and Ethical Issues of Genetic Engineering with Human Beings. (1982): 115. Web. <http://bioethics.georgetown.edu/publications/scopenotes/sn24.htm>.
Thompson, Larry. The First Kids with New Genes. Web. <http://bioethics.georgetown.edu/publications/scopenotes/sn24.htm>.
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