Cri Du Chat Syndrome Analysis

I believe that gene therapy is ethical since it helps those who are suffering from a genetic disorder. Many people who suffer from genetic disorders usually have a lower life span than those who do not and are usually suffering from pain/disabilities due to the genetic disorder. In The BIble there are many instances where God heals those who are sick, the treatment of gene therapy is no different. If doctors are doing what God has been in The Bible why is it immoral? Many genetic disorders have no cure to them and other types of treatment may be ineffective or slightly effective at most. Further research into gene therapy can potentially find a cure to genetic disorders like cystic fibrosis, hemophilia, sickle cell amelia and certain cancers like leukemia and lung cancer. Using gene therapy we able to actually treat and potentially cure genetic disorders since gene therapy corrects the root of the problem; the genes rather than using ineffective treatments or no treatment at all. Some may also say that modifying the gametes of a person is wrong since the kid has no consent whether they chose to receive gene therapy or not, I believe that it is better to apply gene therapy to gametes since it prevents future generations from suffering from the genetic disorder as a whole and it is almost certain the kid will be more thankful not being born with a genetic disorder.

Whether gene therapy should be allowed to be used in practice is currently a very controversial topic. In my opinion, I do not think that it should be allowed. Gene therapy is a technique where genetic material is inserted into a patient to replace missing or defective genes. Gene therapy can be further broken down into two categories: somatic and germline. From there, somatic gene therapy can be approached in two ways: in-vivo and ex-vivo. For the process of gene therapy to begin, the proper vector needs to be acquired. Once a vector is chosen, the vector delivers the new gene into the target cell. Then, the target cell becomes infected with the vector. Next, the vector’s genetic material is inserted into the target cell. Then finally, functional

Cri Du Chat Syndrome arises from anomalies in chromosome five . Cri Du Chat Syndrome is found in boys and girls (Pituch et al., 2010). People from all ethnic backgrounds can have Cri Du Chat Syndrome. Most cases are believed to occur during the development of the egg or sperm. A deletion in the p arm of chromosome five results in Cri Du Chat Syndrome (Tyagi, Kumar, Kumar, Singla, and Singh, 2010). The deletion ranges from a partial deletion to a total deletion on the short arm of chromosome five. The deletion could range from region 5p15.3 to a complete loss of the arm (Rodríguez-Caballero et al., 2010). This abnormality is triggered by a small deletion of a single band of DNA. The genetic information on chromosome 5 is missing. Telomerase

The professionals who wrote this case study made sure to include information pertaining to the abnormalities that were found in most, if not all, of the patients. They

Cri-du-Chat syndrome, or 5p- syndrome is a chromosomal condition where chromosome 5 is missing and occurs in newborn children. The name is a French translation for “cry of the cat” because it sounds just like a cat’s screech. The disease is caused by an abnormal larynx development and usually becomes less noticeable as the child gets older. Since the disease becomes less noticeable through aging, after the age of two, it is very hard for doctors to diagnose a child with it. TERT( Telomerase Reverse Transcriptase), is one of the important genes in cell division that gets deleted because it helps keep the tips of chromosomes in

Cri du Chat Syndrome, also known as 5p- Syndrome, is a chromosomal disorder characterized by severe intellectual and psychomotor development delay commonly associated with total or partial deletion of the end of the short (p) arm of chromosome 5.

On the other hand, the other form of genetic engineering, gene enhancement is the idea of improving average typical genes to be above average. Therapeutic treatment is acceptable, if parents can prevent their child from having a serious or fatal disease, they should be able to pay for genetic treatment if they can afford to do so. However, I completely disagree with the process of genetic enhancement; a parent should not be able to alter their child’s genes from typical or average to above average. If humans even consider gene enhancement for their children, they should revise what is morally and ethically right and wrong. The thought of parents one day being able to enhance or perfect the genes of their expected child is by all means wrong. Children should not be born into a world where their ultimate choices have been made by their parents before the moment of their birth. Children’s genes should be left untouched unless there is something terribly wrong, such as a sickness or disease.

Gene Therapy is an experimental form of treatment that aims to get rid of genetic conditions at their source. It also may be used to replace a faulty gene with a working version or introduce a new gene to cure a condition or modify its effects. ( Victoria State Government, 2011) It is designed to introduce genetic material into cells to compensate for abnormal genes or to create a beneficial protein within the gene. If a mutated or incomplete gene creates a protein needed that is faulty or not present, gene therapy has the ability to introduce a copy of that specific gene to restore the function of the protein within that gene.

Gene therapy has the potential to revolutionize modern medicine. The techniques of gene therapy are still in their infancy as medical treatments and there are still many problems which must be solved before gene therapy will live up to its potential. However, it is very likely that gene therapy will become a reality at some point in the future and when that time comes, the ethical questions surrounding gene therapy will be pushed to the forefront of medicine. Science may find a way to reduce the risks associated with gene therapy but science alone cannot eliminate the serious ethical and societal risks which gene therapy brings to bear on the world. We need to put people's

The first viewpoint is that somatic gene therapy, while risky, is ethical, and germline gene therapy is unethical. Somatic gene therapy can be used to treat cancer, AIDS, cystic fibrosis, Parkinson’s disease, Alzheimer’s disease, Lou Gehrig’s disease, cardiovascular disease, arthritis, or other diseases caused by a single mutation. Changes to a person’s genes in somatic gene therapy do not affect the next generation. They only affect the patient who gave consent for the treatment that could assuage their disease (Gene Therapy, n.d.). Somatic gene therapy can be risky, however, as the DNA could be inserted into the wrong place in the genome (Gene Therapy, n.d.). In rare occurrences, the DNA can incorrectly turn a regulatory gene on or off which can lead to cancer (Gene Therapy, n.d.). While there are rare complications, somatic gene therapy is an overall safe method of treating genetic diseases. In fact, gene therapy to treat sickle cell disease is undergoing clinical trials at Bluebird Bio, the University of California, Los Angeles, and Cincinnati Children’s Hospital Medical Center (Hoban,

Gene therapy works by adding a good copy of a defective gene to the cells in need (“What is”). Gene therapy is different than traditional treatments because it goes right to the source of the problem, rather than just treating the symptoms (“What is”). Although gene therapy is a possible treatment for many medical conditions, conditions affecting many genes is not the best candidate for gene therapy (“What is”). Inserting the gene into a cell can be an extremely difficult process. To insert the gene into a cell, a virus is attached to a vector (Vachani). This vector is used as a carrier and is inserted into the body with the new gene attached. The gene then goes into the cell’s nucleus and becomes attached (Vachani). There are multiple ways to give gene therapy to people and researchers can find the most effective way. Since gene therapy is still new and being experimented with, the side effects are still unknown (Vachani). After the vector is inserted, the body may react and cause a fever, chills, nausea, and a blood pressure drop (Vachani). These side effects are usually gone after a day or two (Vachani). Although it has not happened yet, researchers are worried that the new genes could start to affect the healthy cells (Vachani). Depending on which cells it affects it could

(9) in the future, gene therapy may be approved, not to promote genetic advantage or privilege in offspring, but rather to remediate genetically or otherwise physically inherited genetic diseases and pathological conditions.

A new form of medicine with the potential to save and improve millions of live is gene therapy, or gene replacement therapy. Gene therapy is the use of DNA as a pharmaceutical agent used to treat or cure diseases or disorders caused by defective or missing genes. There are three methods of gene therapy: replacing a mutated gene with a healthy copy of the same gene, inactivating a gene that is not functioning properly, or introducing a completely new gene to help the patient fight a disease. Gene therapy is a promising treatment option, however, the technique has yet to be perfected and is still dangerous and experimental.

Since its inception, gene therapy has captured the attention of the public and ethics disciplines as a therapeutic application of human genetic engineering. The latter, in particular, has lead to concerns about germline modification and questions about the distinction between therapy and enhancement. The development of the gene therapy field and its progress to the clinic has not been without controversy.

Many people question that if gene therapy is existent it would exclude the poor and middle class individuals to perform gene therapy. Discrimination may also be apparent when discussing this technology. Some believe that others without the therapy will discriminate the genetically modified individuals or it may be a vice versa situation. The reaction of society may take two approaches either they will tolerate the situation or be completely intolerant. Moreover, gene therapy may affect our culture and idea in society. The idea of perfectly "modified babies" will certainly affect and arise question regarding our culture and ideal aspect of life. The social impacts involved with gene therapy may be harder to predict due to the change in society periodically, however some theories state some individuals may begin to view this issue more common than others while others would label it as a social or religious custom. Furthermore, since genetic therapy is altering the code of life many people view the issue of altering the genes may be tampering with the nature of life. Another ethical issue, is regarding the diversity in humans. Gene therapy can certainly limit the populations diversity. Moreover, gene therapy can also arise the topic of economics. The rise of gene therapy will strive companies to overcome their competitors that would create competition between the most effective and cheapest development of gene therapy technology. Additionally this will open more job opportunities in the market. Every individual may view the aspects of gene therapy differently therefore decisions of each individual will vary upon their outlooks on this concern