...Gene Therapy SCI115 Introduction to Biology Professor Cassie Prisco November 28, 2014 Gene therapy is a rapidly growing field of medicine in which genes are introduced into the body to treat diseases. It is the name applied to the treatment of inherited diseases by corrective genetic engineering of the dysfunctional genes. It is part of a broader field called genetic medicine, which involves the screening, diagnosis, prevention and treatment of hereditary conditions in humans. The results of genetic screening can pinpoint a potential problem to which gene therapy can sometimes offer a solution. Genes represent the genetic material that organisms pass on from generation to generation. Therefore, genes are responsible for controlling hereditary traits and provide the basic biological code or blueprint for living organisms. Genes produce protein such as hair and skin as well as proteins that are important for the proper functioning of organs. Mutated or defective genes often cause disease. The purpose of gene therapy is to replace a defective gene with a normal copy of the same gene in attempt to restore function. Initially conceived as an approach for treating inherited diseases, like cystic fibrosis and Huntington's disease, the scope of potential gene therapies has grown to include treatments for cancers, arthritis, and infectious diseases. Although gene therapy testing in humans has rapidly advanced, in general, the field of gene therapy has proven to be problematic...
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...Gene Therapy Introduction to Biology 115 Gene Therapy Many medical conditions, disorders, and diseases for centuries went without cures and caused discomfort and even death. Doctors and scientists have worked together regarding many cases in hopes of finding cures for patients and changing the future of modern medicine. The use of gene therapy in the last four decades has been instrumental in many ways. Gene therapy is responsible for the treatment of cancer, diabetes, aids, hemophilia, heart disease, and cystic fibrosis to name a few. Biological Basis In 1865, Gregory Mendel was a fore runner with gene studies which identified the genetic traits that would later lead to revolutionary biological science. Mendel was responsible for providing research for his experiments with plants. The experiments successfully led him to the conclusion that the offspring from mutations could be classified as generational. This theory was correct and laid the foundation for many other scientific studies to eventually lead to significant advancements in genetic study. In the 1960’s scientists exposed the genetic codes which led to decades of discovery to include genetic maps, DNA and RNA sequences, and multiple data strands. Gene therapy was introduced in 1990 with a clinical trial study on a 4 year old little girl suffering from adenosine deaminase deficiency (ADA); which is a severe combined immune deficiency brought on by a mutation of enzymes within a cell. (Gene Med, 2015)...
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...Gene therapy is the transplantation of normal, healthy genes into cells in place of missing or defective ones in order to repair or replace the diseased causing cell, genes. How it works is correct the genetic disorders, like sickle cell, brain cancer and other cancerous cell and viral infections. Biotechnologist have found that there is still no quick rout to achieving this. Gene therapy is still at its experimental stages of treatment, but some of their findings have been promising. The gene therapy starts with a virus. The Genetic engineers then extract the genetic coding from the virus, replacing it with a therapeutic gene that is designed to produce proteins that help fight certain illness. Proteins are then added to the virus. The protein is placed on top of the virus. It then attaches itself to the cell that is causing the disease or virus. This the new genetic code. The new gene with the virus is then injected into a patient or patients. It will then to try get through the body’s immune systems. Sometimes the immune system will attack and destroy the virus before it able to reach the diseased or cancerous cells. The virus only attaches to cells that are compatible to its receptors. When the diseased causing cell starts to respond to the proteins the virus attaches itself to the surface and the cell receptors draws the virus inside this called endocytosis. The virus which contains a genetic coding for enzymes is called integrase. Next the enzyme...
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...Assignment 2: Gene Therapy Science 115 – Introduction to Biology March 1, 2014 Gene therapy is the transplantation of normal genes into cells in place of missing or defective ones in order to correct genetic disorders (Genetics home reference, n.d). This is a promising treatment for diseases such as inherited disorders, some types of cancer, and certain viral infections. The way this treatment works is by introducing genetic material into cells to compensate for genes that are abnormal or are not making beneficial proteins. Mutated genes can cause a protein to be faulty or missing, so a new copy of the gene is introduced to proper function of the protein. This is still a very risky technique and is under study to make sure it will be safe and effective. Currently, it is only being used to treat diseases such as severe combined immunodeficiency—also referred to as SCID or boy-in-the-bubble syndrome—where patients are unable to fight infection and die in childhood (Scientific American, n.d). A further break down of the processes of gene therapy will help to understand exactly how to treatment works. A gene cannot be inserted directly into the cell or it will not function. A carrier called a vector has been genetically engineered to deliver the gene to the cell. This vector can be used in two ways: in-the-body gene therapy and out-of-the-body gene therapy. In-the-body therapy can be injected or given intravenously (through an IV) directly into a specific tissue...
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...Gene Therapy is a relatively new form of treatment, targeted at those who suffer from various debilitating Genetic disorders. It is an advanced type of medicine, and one that is still being trialled, so, at the moment, it is only being used as a last resort in the treatment of certain patients. The aim of Gene Therapy is to give people who have certain genetic malformations the opportunity to combat these, without having to submit to dangerous surgeries or strong drugs. This is a remarkable medical discovery, and holds the ability to heal many illnesses that we label as terminal, like AIDS, cancer and Cystic Fibrosis, increasing sufferers quality of life, if not eliminating the disease entirely. In context, there are 350 million people around...
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...April, 2015 Title? Thesis: While somatic cell gene therapy brings many advantages to the treatment of diseases and the quality of life, using germ-line gene therapy with the opportunities to genetically improve, alter, or fabricate human beings is unethical and should not be researched. Imagine this: You just got back from your germ-line procedure to alter your genes for brown hair and green eyes because you didn't want to pass them on to your children. Everyone is saying that having brown hair and green eyes is unattractive now and they're unwanted traits for future generations. Three months later the “fad” changes and people begin to think that having green eyes won't be so bad after all and that they're actually unique. Now you're left with passing on the Version 1.0 gene pool to your children while everyone else who waited could have children with green eyes. This is the future of germ-line gene therapy. Do we really want to live in a world where this is allowed? The thought of people changing their genes just to fit in and be desirable is unimaginable. While somatic cell gene therapy brings many advantages in the treatment of diseases and the quality of life, using germ-line gene therapy with the opportunities to genetically improve, alter, or fabricate human beings is unethical and should not be researched. Gene therapy is an exciting and new experimental medical procedure that replaces or deletes specific genes in our bodies to either treat or prevent disease....
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...Purpose: The purpose of this article is to describe gene-based therapy and how it transforms the way doctors treat and prevent disease, revolutionize the way we view illness and health and may eventually alter the way we perceive the normative human state. Gene transfer technology will inevitably spread to the world of athletics, bringing with it myriad medical, social, ethical, and philosophical challenges. This article explores some of these issues, with particular attention paid to concepts of harm and privacy. Problem: The problem with gene therapy is that using certain genes like would greatly benefit individuals with anemia and kidney failure or aggressive treatments of cancer but some of these genes are commonly abused in sports. The...
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...in developing effective treatments is an indicator of the efficacy of gene therapy. Haemophilia treatment is effective in curbing the severe bleeding phenotype. Mucus in the lungs resulting from cystic fibrosis has been maintained at the same level instead of further degenerating. The treatment shows a well-tolerated response, but does however need to stimulate a better recovery. Down syndrome has not been treated using gene therapy, but indications of development have been shown with chromosome suppressing technology. Cancer is a likely target for the effective use of gene therapy as an alternative treatment. With acknowledged successes, further investigation can occur...
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...such as the Measles, Polio, and Chickenpox could make the child very ill. Gene therapy is one of many treatments for SCID but has been known to cause Leukemia in a few cases. Is it morally permissible for the researchers to use gene therapy even when they know it may give children Leukemia after treatment? Given that gene therapy has helped save several children lives I believe gene therapy is permissible. What reasons do I have to believe gene therapy is morally permissible? Well according to the theory of Ethical Egoism, the morally right action is the one that produces the most favorable balance of good over evil for oneself. Meaning in every situation the right action is the one that advances one’s own best interests. It’s in the best interest of all to not have SCID and have the chance of getting better and living a normal...
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...Gene therapy is the transplantation of regular/functioning genes to replace abnormal genes. Replacing the defective genes can potentially correct genetic disorders. This procedure is fairly new and illegal in the United States; therefore, causing a controversy in the genetic department of sciences. In the article, “Controversial gene therapy might help women have healthy children” posted in The Washington Post, explains the positives that can come from this experimental solution and also the negative consequences. The focal point of the article is discussing a specific issue with the mitochondria of women’s egg cells. The mitochondria are the energy powerhouses of the cell that synthesizes all of the cell’s adenosine triphosphate (ATP). The...
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...Current research on cystic fibrosis gene therapy suggests that it will become an important treatment strategy INTRODUCTION: Cystic fibrosis is an autosomal recessive disease, triggered by mutation in the gene CFTR i.e. cystic fibrosis transmembrane conductance regulator. CFTR is an ABC gene i-e ATP-binding cassette (transporter) gene that encodes a protein. This CFTR protein is a chloride ion channel protein that controls the flow of chloride ions and water across the cells. This movement is important in generating sweat, digestive juices, as well as mucus secretion and its clearance. This CFTR gene is located at q 31.2 locus of chromosome 7 long arm. The most common mutation in cystic fibrosis is removal of three nucleotides that results in loss of a nucleotide phenylalanine (F) at 508th position at the long arm of chromosome 7. Depending upon the protein expression and function six classes of mutations have been identified in cystic fibrosis: CLASS I: these are non-sense mutations that hinder the protein synthesis as they have premature stop codons. CLASS II: these mutations are most commonly found in patients of cystic fibrosis that include the deletions of phenylalanine 508th del. As a result of this deletion, protein folding is reduced and as a result its transport to cell surface is impaired and it undergoes degradation within the cellular lysosomes. CLASS III: in these mutations, proteins are normally present but are not expressed to a level that responds to intracellular...
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...along with it. I thought it was extremely interesting that we have the same about of genes as a fly. I can see how that would bother some people, but I think it was extremely interesting to find that out. They made a comment like “how could a fly learn how to invent something”. Then someone responded and said “do you know any humans that know how to fly”. That was extremely interesting because that just proved to me that we all have the same amount of genes, but just different genes. Gene therapy was honestly...
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...channel.4,5 The vast majority of mutations involve three or fewer nucleotides and result in primarily amino acid substitutions, frameshifts, splice site, or nonsense mutations.11 Of more than 800 identified CFTR mutations, the 3 base pair deletion of phenylalanine at position 508 is found worldwide in 70% of cystic fibrosis sufferers, therefore making F508 CFTR the most common deadly mutant in the Caucasian populations.6 Since cystic fibrosis has a genetic origin the opportunity to treat by replacing the defective gene with a normal healthy gene (gene therapy) offers a ‘novel therapeutic approach’ for sufferers.7 The estimated survival age of cystic fibrosis sufferers is 33.4 years (Fig 1). In this essay we will discuss the aetiology and symptoms of cystic fibrosis and the current available treatments, with particular emphasis on gene therapy and furanones, which prevent the build up of bacterial biofilms and thus reduce lung infection. Mutations in the CF gene can disrupt CFTR function within epithelial cells in different ways, ranging from complete loss of protein to surface expression with poor chloride conductance.12 The five major mechanisms by which CFTR function is altered are summarized in Figure 2. Class one mutations produce premature transcription termination signals resulting in unstable, truncated, or no protein expression. Class two mutations, including F508, cause the protein to fold incorrectly leading to premature degradation...
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...[pic] Moral and Ethical Issues in Gene Therapy • Dr • [pic] Introduction Genetic research has advanced in a dramatic fashion in the last decade or so, to the point where it has now become possible to attempt therapeutic genetic modification, in a few cases of human genes, where a defects exists which manifests itself in certain serious diseases. This possibility, known as gene therapy, is only in its infancy. At present, no one knows how effective it will prove to be, even in the few conditions on which it is being tried - whether it will only be of relatively limited application, or whether it will open up many wider possibilities. It suffers both over-optimistic claims from some quarters and exaggerated dangers from others, over which the church needs to be discerning. It is, of course, not possible to assert exactly where the possibilities opened up by today's technology will lead in terms of future developments, but various ethical and moral issues are implicit in the technology which it is important to draw to the Church's attention, so that it is forearmed in an area where developments have been taking place at a bewildering pace. An editorial in the "New Scientist" in April 1994 drew attention to the need to weigh up what may still be future issues today, before the technological "horse" bolts from the stable and it is too late to lock the door. Potential Ethical Issues Perhaps the most basic underlying questions centre on a Christian understanding...
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...Gene therapy fact sheet 1. What is a gene therapy? * Gene therapy is an experimental way of using genes to treat or prevent disease. * Gene therapy is an experimental technique for treating disease by altering the patient's genetic material. Most often, gene therapy works by introducing a healthy copy of a defective gene into the patient's cells. 2. Methodology of gene therapy In general, genetic material cannot be inserted directly into a person's cells. Instead, it is delivered to the cells using a carrier, or “vector.” The vectors most commonly used in gene therapy are viruses, because they have the unique ability to recognize certain cells and insert genetic material into them. Scientists alter these viruses to make them safer for humans (e.g., by inactivating genes that enable them to reproduce or cause disease) and or to improve their ability to recognize and enter the target cell. 3. How gene therapy is applied or works As this is still an experimental treatment, the way in which gene therapy is given may vary or change as new techniques develop. Initially, cells are taken from a blood sample. The genes are isolated and changed (engineered) in the laboratory. They are then attached to a chemical or inserted into a vector. Then the changed genes within the carrier are usually given by a drip into a vein (intravenously), through a small tube (cannula) inserted into the vein. They may be given directly into the tumour by injection, which...
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