Human Gene Therapy
Ashanthi Desilva is now able to ride her bike in her suburban Cleveland neighborhood. She can play basketball with her friends or go fishing with her family. She is slightly inconvenienced by her weekly shot of the critical immune system enzyme: adenosine deaminase (ADA). Ashanthi was born with a faulty gene that results in the inability for her body to produce ADA, leaving her dangerously susceptible to even the weakest of infections (Brown). In 1990, researchers collected samples of Ashanthi's blood, isolated some of her white blood cells, and incorporated into them a virus engineered to contain a healthy ADA gene. These cells were then injected back into her body. She was the first patient to undergo gene therapy, and without that therapy and the weekly shots of ADA, she would have been destined for a life of isolation.
Human gene therapy is the replacement of an absent or faulty gene with a functioning gene. As a result, the body is able to produce the correct enzyme or protein, thereby eliminating the cause of the disease (Gene_Therapy_Overview). There are essentially two types of gene therapy: somatic cell therapy and germ line therapy. Somatic cell therapy involves treating any cells of the individual, except the gametes, at the cellular level to correct an absent or malfunctioning gene. This can be accomplished in three ways: ex vivo, in situ, or in vivo. Ex vivo involves removing cells from the patient, altering the genetic material, and placing them back into the patient. In situ requires the vector be placed directly into the affected tissues. In vivo gene therapy involves injecting the vector into the bloodstream. The vector then must find the target tissue and deliver the therapeutic genes. Germ line gene therapy treats the gametes or an embryo, which would be used in the case of in vitro fertilization. The difference between somatic and germ line gene therapy may seem to be subtle; however, the alterations obtained through germ line therapy are not only found in that generation, but are passed on to the individuals progeny. That has serious repercussions when it comes to discussing the ethics of using germ line therapy.
Presently, similar techniques are being examined for both somatic and germ line gene therapy, but germ line therapy is more difficult (Coults). This is not to say that somatic cell gene therapy is easily accomplished. One challenge facing researchers is finding a suitable vector that would safely and efficiently deliver the genetic payload into the patients' genome.
Germline gene therapy is where the correct "good" gene is inserted into the germline in place of the defective "bad" gene, and when reproduction occurs the gene will be passed on to the progeny. Inserting the "good" gene into the very early embryo sta ges of development allows for both germline and somatic cells to be corrected. Government has limited the research to only somatic cell gene therapy such as performed in Cystic Fibrosis research.
The years leading up to the 1972 election were filled with new political tactics. Going into the election year, President Nixon seemed like he could never lose the second term election after successfully negotiating with Vietnam, Beijing, and Russia to improve international relations (Emery 4). Raising international toughness made Nixon seem like the most worthy person to stay president. Fred Emery analyses in his novel Watergate: The Corruption of American Politics and the Fall of Richard Nixon, the president was also setting up the first summit meeting in history with Soviet Union Presidents (3). There seemed to be nothing capable of holding the seemingly responsible man back. However, this assurance came with massive consequences. The absolute certainty that Nixon would be reelected fueled the lies and abuse of power by the Nixon government (Emery 195). As the outlook of landslide winnings took over the White House, the moral reasoning, “the end justifies the means” became more prevalent. Nixon was obsessed with winning and being successful. Under his command his staff did whatever possible to ...
...re than the upper; in some cases, only the lower extremities are involved. The last category of Spastic CP is quadriplegia only 10-15% have it and it affects all 4 extremities as well as the body. Extrapyramidal or dyskinetic cerebral palsy affects 10-15% common characteristics of this type of CP are often slow uncontrolled jerking motions. It may affect hands, feet, arms, and legs and in some rare cases the tongue and face. Ataxic Cerebral Palsy (CP) is rare only a small percentage about 5% of all total cases of CP have Ataxic CP . It affects the sense of balance and coordination as well as quick movements or movements that require control such as writing. Affected persons might have a hard time controlling legs and may often walk unsteadily. Mixed CP is generally a mixture of any of the other CP generally it divides patients into mild, moderate, and severe types.
The renal disease are common nowadays .The acute renal failure is a medical term means that the kidneys stopped from working and not able to clear toxins from body ,not able to maintained a stable electrolyte balance inside the body and not able to secret the extra fluid as urine outside the body. The renal replacement therapy (RRT) or dialysis has been discovered on 1913 by Able, Rowntree and Turner in London, UK.
Gene therapy works in three ways; it works to replace a missing or defective gene with a normal one, replace a faulty gene so that it will function properly and it works to activate and deactivate a gene, allowing it to “switch” on and off. Gene therapy is done by the deliverance of a gene to a cell via a carrier, or vector, such as a virus. Scientists lean more towards using a virus because they can seek out particular cells and transfer pieces of deoxyribonucleic acid into them. Scientists also take advantage by deactivating their harmful characterizes and modifying them to carry particular gene into designated cells. After gene therapy is done, the genes can then stimulate the production needed for standard functioning, allowing that gene to return to its previous normal state. Therefore, if a patient were to be in the beginning stages of cancer, gene therapy would seek out the cancerous gene and replace it with a healthy one and minimizes the disease from
In September 14, 1990, an operation, which is called gene therapy, was performed successfully at the National Institutes of Health in the United States. The operation was only a temporary success because many problems have emerged since then. Gene therapy is a remedy that introduces genes to target cells and replaces defective genes in order to cure the diseases which cannot be cured by traditional medicines. Although gene therapy gives someone who is born with a genetic disease or who suffers cancer a permanent chance of being cured, it is high-risk and sometimes unethical because the failure rate is extremely high and issues like how “good” and “bad” uses of gene therapy can be distinguished still haven’t been answered satisfactorily.
It was a treatment for a four-year-old girl named Ashanthi DeSilva, who were born with an adenosine deaminase (ADA) deficiency, an autosomal recessive disorder that affect the immune system. Her doctors genetically modified her defective immune cells to function as normal ones. Then, they used a virus that also had been genetically modified to remove its harmful genes to deliver the corrected immune cells back to her body. This early success led to many other gene therapy trials in the 1990s for different kinds of genetic diseases, until a tragic setback happened. In September 1999, Jesse Gelsinger became the first person who died after undergone a gene therapy for ornithine transcarbamylase (OTC) deficiency, a rare metabolic disorder. He died from massive organ failure caused by a bad reaction of his immune system to the virus used in the therapy (Thompson
When concerning the home front of 19th century Europe, women were “the cult of domesticity” and were highly regarded as wives, mothers, and part of the working class. A lofty character was necessary in completing the demanding tasks surrounding the home life. Images of women ranging from newspapers to fine art all displayed the univ...
In this paper, I will argue that genetic therapies should be allowed for diseases and disabilities that cause individuals pain, shorter life spans, and noticeable disadvantages in life. I believe this because everyone deserves to have the most even starting place in life as possible. That is no being should be limited in their life due to diseases and disabilities that can be cured with genetic therapies. I will be basing my argument off the article by “Gene Therapies and the Pursuit of a Better Human” by Sara Goering. One objection to genetic therapies is that removing disabilities and diseases might cause humans to lose sympathy towards others and their fragility (332). However, I do not believe this because there are many other events and conditions in society that spark human compassion and sympathy towards others.
"The aim is to decrease the fear of a brave new world and to encourage people to be more proactive about their health. It [Gene therapy] will help humans become better physically and even mentally and extend human life. It is the future” (Hulbert). Dr. Hulbert, a genetic engineer, couldn’t be anymore right; more time, money, and research needs to be put into gene therapy and genetic engineering, since it can cure certain illness and diseases that are incurable with modern medicine, has fewer side-effects than conventional drugs or surgery, and allows humans to be stronger physically and mentally at birth. Gene therapy or genetic engineering is the development and application of scientific methods, procedures, and technologies that permit direct manipulation of genetic material in order to alter the hereditary traits of a cell, organism, or population (NIH). It essentially means that we can change DNA to make an organism better. Genetic engineering is used with animals and plants every day; for example with genetic...
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. Although initially considered as a promising approach for treating the genetic of disease, the field has attracted disappointment for failing to fulfil its potential. With the resolution of many of the barriers that restricted the progress of gene therapy and increasing reports of clinical success, it is now generally recognised that earlier expectations may have been premature.
Types and Forms of Cerebral Palsy. (2007). My Child 2007. Retrieved December 2, 2013, from
Gene therapy gives patients who born with diseases that are incurable to traditional medicine a permanent cure. If patients received germ-line therapy, which involves replacing disordered genes in sperms or eggs, their offspring would also have correct gene orders. The positive effect would influence the whole family.
Advancements in science and medicine are usually accompanied with a myriad of ethical and moral implications. The fairly recent advancement in genetics called gene therapy is no exception to the baggage of polarizing views that come with new technology. Gene therapy is an extremely hot topic in both the science world and everyday life. New technology, discoveries, and breakthroughs are rapidly occurring in the field every day. The topic of gene therapy in humans is one that is highly debated due to the ethical implications connected to the science. Both sides of the debate have various reasons for their position, but the main factors come down to the ethics of changing someone’s genome and the consequences that accompany the altercations. The two types of gene therapy, somatic and germ-line are seen in different lights. There is more debate over germ-line therapy because the alterations have more consequences than somatic gene therapy. There are many moral and ethical decisions that need to be considered before gene therapy can be widely accepted. Do we have the right to change a person’s genetics, especially before they are born? Do we know enough to confidently insert or delete genes without detrimental consequences down the road? If we have the ability to help people who have disabilities or diseases, is it ethical to withhold and not treat the patient? I believe human gene therapy is a good and useful tool for medicine and needs to be developed because it posses the ability to help and cure people from ailments that degrade their quality of life.
Retro-viruses and gene-specific medications could be used to alter a person’s genetic code, ridding a person of inherited maladies such as heart disease or diabetes. With the introduction of some, if not all, of these different methods of treating ailments, we could effectively wipe out a large amount of diseases that would otherwise be untreatable. Senior citizens would no longer have to suffer from maladies such as Alzheimer’s or other such illnesses related to age. With these procedures, a child can grow up never having to suffer from a learning disorder such as ADHD (Attention Deficit Hyper-activity Disorder) or even cases of mental retardation by eliminating or modifying the genes that are responsible for these and other problems.