History And Procedures of Gene Therapy
explanatory Essay
1810 words
The History And Procedures of Gene Therapy
Abstract: Over the course of history there has been the idea of gene therapy has inspired many great scientists. The history of eugenics is important to the history of gene therapy because it is how gene therapy originated. Eugenics has driven many people to take extreme measures to try and make a “better human race”, this includes the Nazi party and the movement in the 1930’s inspired by Francis Galton. After that, research in eugenics continued and the human genome project sprung from the minds of scientists. After the inspiration of the human genome project scientists developed theories that gene therapy is possible. After a series of experiments that then failed, scientists were discouraged and the future of gene therapy is now being doubted.
Gene therapy is an idea that has fascinated humans and scientists for centuries. Gene therapy theoretically can allow scientists to change the human genome and peoples DNA. Ideally, scientists wish to replace “bad genes” in your DNA that can potentially hurt you with good genes that will help you avoid any genetic ailment. In fact, all that scientists really want to accomplish is to replace our natural genes with unnatural ones. The idea and concept of gene therapy is not new to human history. On the contrary, gene therapy is a concept that has been fascinating the minds of many scientists and researchers over the span of many centuries.
Although the idea of genes was first born during the times of Plato, the concept of gene therapy wasn’t really born until the late 1900’s. Mr. Francis Galton, who is the younger first cousin of Charles Darwin, had the idea that the human race could be improved according to the method of selective breeding....
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...The Last 80 Years in Primary Immunodeficiency: How Far Have We Come, How Far Need We Go” Shearer, William & Fischer, Alain Department of Pediatrics and Immunology, Baylor College of Medicine, and the Department of Allergy and Immunology. February 9, 2006. Accessed June 17. http://www.sciencedirect.com/science?_ob=ArticleURL&_udi=B6WH4-4JS81T0-M&_coverDate=04%2F30%2F2006&_alid=425456057&_rdoc=1&_fmt=&_orig=search&_qd=1&_cdi=6840&_sort=d&view=c&_acct=C000059598&_version=1&_urlVersion=0&_userid=4421&md5=b273338ae3c563bfa35380a2e42aff41
United States. Environmental Protection Agency. Office of
Emergency and Remedial Response. This Is Superfund .
Jan. 2000. 16 Aug. 2002 superfund/whatissf/sfguide.htm>. Lyon, Jeff & Corner, Peter. Altered Fates: Gene Therapy and the Retooling of Human Life. W. W. Norton & Company Inc. New York, New York. P. 1995.
In this essay, the author
- Explains that eugenics is important to the history of gene therapy because it is how it originated.
- Explains that gene therapy is an idea that has fascinated humans and scientists for centuries. it allows scientists to change the human genome and people's dna.
- Explains that although the idea of genes was first born during plato, the concept of gene therapy wasn't really born until the late 1900's.
- Explains that eugenics became popular among the upper class and middle-class whites in the united states, britain, and germany. they wanted to rid their society of "blight" by setting up a breeding program that excluded genes that could cause violent behaviors and conditions.
- Explains that eugenics was used for less than good intentions during the reign of adolph hitler. they hoped to eliminate color and make just one completely caucasian race.
- Explains that recombinant dna is the material in an organism that regulates how that organisms body functions. it is made up of amino acids, sugars and phosphates.
- Explains that recombinant dna is the basis of gene therapy, which is taking a piece of dna and replacing, inserting, or extracting another piece.
- Narrates how robert sinsheimer and charles delisis collaborated on the human genome project in the 1980s.
- Explains delisis' plan for a five-year doe human genome program that would include mapping, development of automated high-speed sequencing technologies, and research into computer analysis of sequencing data.
- Explains that several countries in europe were gathering momentum researching the human genome during 1987-1988. scientists were convinced that the project was the key to understanding life.
- Explains that the human genome project was established to analyze human dna and understand what the dna codes for and what results from that coding.
- Opines that scientists have done many experiments hoping for success, but have been disappointed about the progress that has been made in using gene therapy for medical reasons.
- Opines that the fda declared that gene therapy was too dangerous to pursue without extensive security and safety issues.
- Concludes that gene therapy has been misunderstood and even given a bad name, due to mistreatment by nazi parties during world war ii and the holocaust. curiosity on the subject of eugenics and dna led to the human genome project.
- Opines that gene therapy is the cure to never getting sick again and the ultimate medical treatment, but there have been some discouraging outcomes and disappointment considering the subject.
- Explains that boy’s cancer prompts fda to halt gene therapy.
- Explains shearer, william & fischer, alain department of pediatrics and immunology, baylor college of medicine, and the department
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- Gene Therapyexplanatory essayGene Therapy Gene therapy is the term used for the process of curing various diseases on the genetic level by injecting patients with a healthy gene so their bodies will continue to produce healthy copies of the gene rather than destructive ones. The term gene therapy and the basic notions that it entailed weren't even in researchers' vocabulary until the mid 1960's. Early endeavors to cure people via a genetic approach began in 1970 with a disease called argininemia, but proved ineffective. An actual effective transfer of a gene didn’t occur until nine years later in 1979. Early attempts to examine the ethical nature of the future of gene therapy stemmed from this initial successful experiment and ones immediately following it.2 Throughout the 1980’s and early 1990’s development of gene therapy continued.
In this essay, the author
- Explains that gene therapy is the process of curing various diseases on the genetic level by injecting patients with a healthy gene so their bodies will continue to produce healthy copies of the gene.
- Describes the development of gene therapy in the 1980s and early 1990s. jesse gelsinger's adenovirus vector was the first clinical trial.
- Describes jesse gelsinger's death from a gene therapy experiment targeting the gene for the rare genetic disorder ornithine transcarbamylase.
- Explains that the study was performed by the university of pennsylvania's gene therapy researchers. jesse was the 18th subject to undergo the treatment.
- Describes how jesse's immune system over stimulation led to blood clotting, hemorrhaging, an increase in temperature, lung failure due to excessive white blood cells, and a dramatic rise in ammonia levels.
399 wordsRead More - Gene Therapyexplanatory essayGene Therapy The Human Genome Project began around 1986. The main goal of the project is to locate and sequence all genes found in human DNA. The objectives for this ambitious effort are to learn more about heredity of disease and to discover the genes that would aid in gene therapy. Advances in gene therapy strive to treat hereditary diseases and possibly eliminate disease from the genome.
In this essay, the author
- Explains that the human genome project provides assurance that children will be born healthy and live long healthy lives. a drop of blood from the developing fetus or a schwab from inside the child's mouth would acquire the necessary cells.
- Explains the advantages of gene therapy, such as the production of interferon, which is produced by cells in the human body in response to viral attack.
- Opines that we were given the ability to better the human race by gaining the knowledge of how to use gene therapy.
- Cites wilson, jim. the institute for human gene therapy.
- Describes the human genome project, which aims to locate and sequence all genes found in human dna. advances in gene therapy aim to treat hereditary diseases and possibly eliminate disease from the genome.
- Explains how gene therapy began with the human genome project, which has found gene locations for many diseases.
- Explains that technology is making it easy to find out a person's genome. there are cases where insurance companies have obtained the information and then refused to insure the individual.
- Explains elmer-dewitt, philip, and grace, eric s. the genetic revolution. time, january 17, 1994.
1624 wordsRead More - Gene Therapyexplanatory essayGene Therapy: A new generation of molecular medicine In September of 1990, eight year old Ashanthi DeSilva made medical history when she received the first authorized human gene therapy treatment. Ashanthi has been born with a defective gene that normally produces an essential enzyme adenosine deaminase (ADA). If left untreated the inability to produce this enzyme results in the fatal malfunction of the immune system.
In this essay, the author
- Explains that ashanthi desilva received the first authorized human gene therapy treatment in 1990. she was born with a defective gene that produces an essential enzyme, which causes the immune system to malfunction.
- Explains the technical and ethical aspects of gene therapy and a synthesis of 's personal opinion.
- Explains that rdna technology has made the transfer of genes from one organism to another possible. gene therapy involves augmenting the functions of an absent or dysfunctional gene by the introduction of a functional gene into the cells of the individual.
- Explains that germinal gene therapy involves the introduction of genes into both somatic cells and the germline of an individual.
- Explains somatic gene therapy, which focuses on the correction of genetic disease by treating non-reproductive tissues. it involves the removal of dysfunctional cells and inserting a cloned wild-type gene.
- Explains that there are two types of gene delivery vehicles, non-viral and viral. non-viral delivery includes approaches such as direct injection or mixing the dna with compounds that allow the gene to cross the cell membrane.
- Explains that retroviruses are rna viruses that convert their genome into dna in the infected host cell.
- Explains that adenoviruses are a family of dna viruses that can infect both dividing and non-dividing cells.
- Explains that adeno-associated viruses are non-pathogenic single stranded dna viruses that contain relatively few genes and require additional genes to replicate.
- Explains that the evaluation of gene therapy as a treatment for genetic disease has begun in the united states. many trials have resulted in short-term improvement in patients receiving treatments, but effects have not been sustained.
- Analyzes how the media's influence has shaped public views of genetics, and science as a whole. gene therapy has not been an exception and the following sections address some of the issues that have emerged during recent years regarding the ethics and potential of this technology.
- Explains that genetic diseases have been expanded to include any trait that has a genetic component even though the heritability is reduced.
- Explains that the safety concerns of gene transfer that are required for gene therapy to be successful are not trivial. patients must risk vector-induced inflammation and immune responses.
- Opines that gene therapy holds both great promise and potential for misuse. the present state of the technology is irreversible and should not be approached without prudent and judicial caution.
- Opines that potential misuse of gene therapy is undeniable, and concerns about it being used for enhancement of desirable qualities such as intelligence, cosmetic appearance, or physical performance need to be addressed.
- Opines that the media has played a pivotal role in the public perception of what the goals of clinical trials truly are.
- Explains that scientists have shown a sense of moral and social responsibility since the alisomar conference in 1974. committees and government agencies have played an important role in the evaluation of the ethics of recombinant dna technology.
- Opines that a renewed emphasis in basic research is needed to sustain these efforts. researchers must address shortcomings in critical components of virology, immunology, and cell biology if gene therapy is to advance.
- Explains that the recombinant dna controversy is twenty years later, and that eugenics is the narrowing of normality.
- Explains the bystander effect in gene therapy: great, but how does it work? the journal of nih research.
- Cites magnus, d., marshal, e. and msezane, l.
2351 wordsRead More - Gene Therapyanalytical essayGene Therapy I. Introduction With the human genome project now completed, identifying our DNA, the next step forward is being taken to analyze this information and apply it in a helpful context. As we discover which genes affect and trigger the different traits humans possess, new questions result pertaining to potential problems in our DNA as well as genetic enhancement opportunities. In theory, once a problem is pinpointed within a person’s DNA, there exists a possibility for correcting this defect.
In this essay, the author
- Explains that gene therapy is a technique used for correcting defective genes responsible for disease development, which sparks controversy when societal implications are examined.
- Explains that gene therapy is a relatively new field that leaves much open to be explored.
- Explains that genetic enhancement offers the power to enhance normal functions, much like a type of molecular cosmetic surgery.
- Analyzes the different facets of gene therapy, including somatic, germ-line, and genetic enhancement. ethics guide decisions and actions based on duties derived from core values.
- Opines that the future use of gene therapy should proceed with caution and with an attitude of awareness.
- Cites adams, harry, miller, fins, and fitzgerald.
- Cites silva, fred g., thompson, richard e. and williams, d.
2631 wordsRead More - Treating Genetic Disorders with Gene Therapyexplanatory essayGene Therapy With our current society rapidly advancing in both the technological and medicinal world, many new treatments have been born that can be used to treat genetic conditions that regular antibiotics and surgeries simply have no positive or progressive effect. Gene therapy is when a newly developed gene is transferred, or introduced, to an already existing gene, it is the attempt to treat one’s genetic disorder at the molecular level and could significantly improve the human population and provides hope to people with disabilities. Gene therapy goes straight to the source and alleviates symptoms of the disorder. The gene receiving this new development is in some shape or form, mutated, or defective.
In this essay, the author
- Explains that gene therapy is the attempt to treat one's genetic disorder at the molecular level and provides hope to people with disabilities.
- Explains that gene therapy begins with locating the dysfunctional gene. the most complicated part is the transportation of a new gene into another.
- Explains that a vector must be able to target the right cells, integrate the gene in the cells and successfully become part of the host cell.
- Explains that gene therapy has both positive and negative outcomes, including the treatment of hereditary blindness, immune deficiencies, hemophilia, blood disease, cancer, and parkinson’s disease.
- Explains that human gene therapy is not yet accepted as a full-functioning treatment because there are still risk factors to consider.
- Explains that if a patient is treated for genetic disorder, it can still be passed down to their offspring unless the egg and sperm cells are targeted by the same treatment as well.
- Explains that gene therapy is controlled by the fda, the food and drug administration, and the national institute of health.
- Explains that the first patient to receive gene therapy was a 4 year old child with sever immunodeficiency disease. the girl's white blood cells were extracted and exposed to the new genetically engineered virus.
- Explains the success story of boy-in-the-bubble syndrome, which is a severe combined immunodeficiency that leaves patients at the mercy of infections and often dies in their early childhood. the most impressive is the recent advancement in gene therapy for blood cancer treatments.
- Opines that gene therapy should be more researched and looked into and especially should have more support. it has risks, but diseases have casualties.
- Describes the resources available at http://www.ndsu.edu/pubweb/mcclean/plsc431/students98/fleck.htm.
1180 wordsRead More - The Pros And Cons Of Cancer And Gene Therapyexplanatory essay... fight the disease. It is crucial that regulation be a necessary component of gene therapy research and applications. In hopes that the government can regulate and can receive this treatment, not restricting it to people that has serious genetic diseases. Gene therapy will change the field of medicine from what it is today. As scientist discovers more genes and their functions, the potential of this treatment is limitless. Though gene therapy is an auspicious treatment choice for numerous diseases (including inherited disorders, some types of cancer, and certain viral infections), the procedure remains precarious and is still under study to make sure that it will be safe and effective. Thus government regulators and scientist must take a lead role in adopting a practical approach to address these issues and determining the correct procedures for dealing with them.
In this essay, the author
- Explains that gene therapy is a process of replacing defective or undesired genes in the body with "normal" genes. gene therapy can be done ex vivo by taking cells from patients bone marrow or blood.
- Explains that gene therapy involves inserting human genes into a bacteria cell, then translating the information into protein, and then introducing the protein into human cells. major advances in recombinant dna technology have occurred over the last 20 years.
- Describes how gene therapy was carried out on a four-year-old girl, ashanti desilva, who was born with an autonomic recessive metabolic disorder.
- Explains that germinal cell gene therapy involves the genetic modification of germ cells and would be carried on to the future generation.
- Describes somatic cell gene therapy as the transfer of a gene or genes into body cells other than germ (egg or sperm) cells with effect only on the patient.
- Explains that somatic gene therapy is a type of genetic therapy that targets specific areas. it has caused technical and moral conflicts, especially when human embryos are used.
- Explains that when using gene therapy as a technique, the cell in the body and how it uses this gene must be noted.
- Explains the use of "engineered viruses" to insert genes into target cells. these viruses are inactivated so that they can pick up and transport genes of choice.
- Explains that the best way to get genes into cells is by using disabled and engineered viruses. adenoviruses are known to be the largest viruses that do not have an enveloped membrane.
- Explains that cancer causes cells to grow aberrantly, causing damage of normal tissues, loss of function, and pain. multiple cumulative mutations are needed to cause cancer.
- Explains the effect of betulin and its acetylenic derivative on production and apoptosis in human melanoma cells.
- Explains that gene therapy research involves modifying viruses so that instead of introducing their own genes, they introduce a therapeutic gene which will cure the patient's disease.
- Explains that the second major type of vector is based on the retrovirus, murine leukemia virus (mlv). gene activity is easy to control and continues over long periods of time.
- Explains that most of the research in genetic therapy is more concerned with the treatment of cancer.
- Opines that gene therapy is an experimental method that in particular targets genes to prevent diseases, but opponents, biomedical reductionism, will undoubtedly have several responses.
2435 wordsRead More - Ethics of Genocide and Eugenicsanalytical essayJust as there are different types of people who look at one glass of water and describe it as half full or half empty, the public has many different views on the future of our society. Gene therapy is also a glass that can be viewed in different angles – different perspectives. Some say it has great potential to shape the ideals of our future, while others believe it signifies intolerance for disabilities, imperfections that supposedly deplete from a person’s interests, opportunities and welfare (quoted by Peter Singer, xviii). This global issue has brought people with different opinions in the open, arguing their views using history, morality and foresight.
In this essay, the author
- Explains that the public has many different views on the future of our society. gene therapy is a glass that can be viewed in different angles – different perspectives.
- Analyzes how success and perfection play a role in determining human characteristics, once thought inalterable. parents want their children to be born with an opportunity to excel.
- Argues that society fails to recognize the social value of future persons with disabilities and conveys a devaluation of those now living with disability.
- Argues that scientists are demeaning the lives of those presently living with genetic disorders by searching for a cure for them.
- Argues that gene therapy is a win-win strategy, since it replaces defective genes with normal ones to cure genetic disorders.
- Argues that somatic gene therapy is more or less ethical than germline, but it has not been applied anywhere except in the lab experiments.
- Describes ethnic groups and deaf peoples, and explains that they are distinctive in terms of common cultural traditions or heritage.
- Argues that gene therapy could lead to a future where everyone starts life with an equal chance of health and success.
1413 wordsRead More - The Ethics of Gene Therapy: Balancing the Risksopinion essaySince 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.
In this essay, the author
- Proposes a set of principles and practical measures that might assist both researchers and patients, to enable more informed decisions about risk.
- Explains the limitations of existing treatments, including availability and associated risks, are the starting points for risk analysis and influence the levels of acceptable risk in gene therapy trials.
- Explains that the frequency and magnitude of gene therapy risks in humans are unknown. this uncertainty arises from a lack of knowledge about types and frequency of adverse events, but it is also reducible as knowledge is accrued during clinical trials.
- Explains that balancing the risks of gene therapy research requires analysis of the possible benefits. clinical research is intended primarily to benefit society by furthering knowledge that can be implemented for the therapeutic benefit of future generations.
- Explains that patients perform an assessment of risks when deciding whether to consent to a clinical trial. the attitudes and values they bring to this risk assessment may usefully inform researchers' and iecs' normative judgments.
- Opines that qpls could help patients balance the risks and benefits of gene therapy with those of conventional treatment.
- Asks the gt societies to develop their own standards/guidelines and post them on websites for use by their members.
- Opines that the development of the gene therapy field and its progress to the clinic has not been without controversy.
- Argues that risk assessment in clinical research is an intrinsically difficult task due to the extent of uncertainty involved.
- Opines that when there is no data on gene therapy risks in humans, it may be considered acceptably low if researchers have no reason to think they are higher in frequency or magnitude than the known risks of conventional treatment.
- Opines that models based on statistical methods to account for epistemic uncertainty may provide useful guidance during risk assessment.
- Argues that a mechanism of peer review by independent experts in the field would strengthen the societal benefit of trialling that protocol in humans.
- Opines that objective evaluation of scientific merit of clinical trial protocols from independent experts would strengthen the societal benefit of research.
- Explains that the possibility of benefit to individual patients is a relevant consideration during risk assessment. research is intended primarily to benefit society through the generation of knowledge.
- Argues that patients' attitudes towards risk may help researchers and iecs make the value judgments that risk assessment involves.
- Opines that effective communication of risk information is essential to empowering patients to make informed risk assessments and hence decisions about consent.
2583 wordsRead More - Muscular Dystrophyopinion essay"Gene Therapy--Promises, pitfalls and prognosis", The New England Journal of Medicine (September 28, 1995, Pg. 871-873)
In this essay, the author
- Explains that medicine, biology, and health care have become a primary concern of society, while daily programs such as cnn's your health have brought the science and debates around it to the center of our society.
- Explains the term muscular dystrophy, which is used generically to encompass several muscular disorders present at birth and probably all genetically inherited. there are at least six major forms of the disease.
- Explains that most boys with muscular dystrophy have a gait disturbance before they turn 3. the symptoms are dismissed as simple clumsiness, but the disease progresses to the point where the children have difficulty rising from the floor.
- Explains that duchenne muscular dystrophy affects 1 per 3,500 male births worldwide. the cause of death is not always clear, but respiratory insufficiency and heart failure are contributing factors.
- Explains that duchenne and becker muscular dystrophies are variable phenotypic expressions of a gene defect at the xp21 site.
- Explains that duchenne muscular dystrophy is inherited in an x-linked recessive way although in up to a third of cases it occurs in families with no previous history of the disease.
- Explains that dna markers from amniocytes are error ridden and deletion mutants detectable in only 65% of cases. however, there is new hope for women in families with dmd.
- Opines that the media has given a lot of attention to gene therapy, while critics say that man is being too arrogant and this will ultimately lead to his demise.
- Explains that two different approaches have been developed in the pursuit of a cure for dmd. the first and oldest form is that of myoblast injections.
- Explains that the most promising way of curing muscular dystrophy is by shuttling the dystrophin gene to the muscle cells through retroviruses.
- Opines that successful gene therapy for duchenne's muscular dystrophy and cystic fibrosis requires the delivery and long-term expression of the appropriate gene to large numbers of cells throughout inflamed and
- Argues that insurance companies and consumers will have many differences when technology is fully available. they argue that denying coverage and/or modulating how much each has to pay based on his genetic profile violates the basic covenant of insurance.
- Opines that muscular dystrophy has brought people together in a world where people tend to compete against each other and divide.
- Analyzes how a society needs these differences to put people's lives in perspective. these diseases compel us to think about ourselves and discuss what it means to be human beings.
- Opines that when gene therapy for is developed for md will humans be able to stop the wave of other therapies that use the same technology?
- Opines that a cure for muscular dystrophy would end pain and grief. insurance companies cannot dictate people's lives in order to reach financial targets.
- Opines that it is difficult to discuss and fathom such profound issues and dilemmas in an essay, but the true challenge is to unite the science with the ethics.
- Opines that there are good reasons to be optimistic about the ultimate success of gene therapy. the next step is to create the correct framework for this revolution in molecular medicine.
- Cites the columbia university college of physicians and surgeons, complete home medical guide, and gerald m. fenichel, clinical pediatric neurology.
- Cites emery, alan e. h., duchenne muscular dystrophy, science news, the economist, new york times, and wall street journal.
2877 wordsRead More - Human Gene Therapyexplanatory essayHuman gene therapy Gene therapy is a technique used in attempts to cure or prevent genetic diseases at the molecular level (basically at the source) by correcting what is wrong with defective genes, a good version of the defective gene is introduced into the existing cells. This technique is still considered experimental, only being done through clinical trials. This idea was first suggested during the 1950’s. The basic idea was “if the basses can be arranged incorrectly then why can’t they be rearranged in the correct way?”
In this essay, the author
- Explains that the ethics surrounding human gene therapy produces a portion of the controversy surrounding it.
- Explains what a genetic disease is. advocacy, education & empowerment.
- Opines that gene delivery: tools of the trade. web. 19 may 2014.
- Explains that bose, debopriya, "gene therapy pros and cons." buzzle.
- States that herscher, elaine, "fighting diseases with genetic therapy." los angeles times, 13 sept. 2012.
- Describes gene therapy as a technique used in attempts to cure or prevent genetic diseases at the molecular level by correcting what is wrong with defective genes.
- Opines that gene therapy needs more work to perfect it, but it may never have a 100% success rate. genetic screening is important for research and could cause problems among the public.
1684 wordsRead More