...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 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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...reality. Fifty years later in 2003, National Institute Health accomplished mapping of 32,000 genes of the human genome. Gene mapping was a significant research that enabled researchers to focus on gene specific diseases and birth defects. More than 1000 research projects started inward voyage discovery of human kind rather outward exploration of the planets. Clinical research involving human beings have greater potential for risk of misusing the technology. Potential for misusing the genetic research and fear of unknown long term effects on the successive generations have sparked bioethics debates. Like abortion, gene mapping is a very controversial subject and both sides have strong views. For example, should a woman abort the pregnancy based on the abnormal genetic screening results? Genetic technology provides an enormous power within our grips and with this enormous power comes following the ethical standards. Ethical standards should be followed involving human subjects. Genetic screening standards should align with four ethical principles of: • Respect for the human autonomy, dignity, and obtaining informed consent • Genetic screening should cause the minimal amount of harm to the subject • Provides maximum benefit to the subject • Genetic screening should be available to all the patients of a certain population considered high risk for a certain disease or condition Purpose of this paper is to discuss the correlation between genetic screening, four ethical principles...
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...DNA Technology DNA research has come a long way since Friedrich Miescher first isolated it in 1869 and the discovery of DNA’s double helix structure by James Watson and Francis Crick in 1953. Surely they would have never imagined what scientists can do with human DNA today. Cloning, gene therapy, stem cell research, and genetically modified foods all began with the discovery of DNA and probably would have been unimaginable in anyone’s eyes in the 1800’s. But scientist’s today are manipulating genes and DNA in an effort to prevent disease, cure disease, and feed the world. History was made on July 5, 1996 when Dolly the sheep was born. She was the first mammal to be cloned from adult DNA. By splitting two-cell embryos apart, scientists were able to produce two genetically identical organisms. Cloning is a process of making genetically identical organisms through non-sexual means. There are three types of cloning, DNA cloning, reproductive cloning, and therapeutic cloning. DNA (or molecular) cloning consists of removing a small piece of the DNA strand and uniting it with a plasmid which reproduces itself to create multiple copies of the same DNA code. The copied DNA can then be grown in a suitable host cell where the recombinant vector can then be reproduced along with the host cell DNA. DNA cloning is typically used in biological experiments and technological applications where large scale protein production is needed. (NRC, 2002) Reproductive cloning...
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...Genetic Engineering Research & Position Paper Table of Contents DNA Fingerprinting/ Genetically Modified Foods --------------------------------------------------- p. 3 Gene Therapy/Cloning------------------------------------------------------------------------------------ p. 4 Stem Cell Technology------------------------------------------------------------------------------------ p. 5 Position Paper------------------------------------------------------------------------------------------- p. 6-7 Bibliography-------------------------------------------------------------------------------------------- p. 8-10 Evaluation Rubric----------------------------------------------------------------------------------------- p. 7 DNA Fingerprinting DNA fingerprinting are tests that help to identify and analyze a person’s DNA. The term “fingerprinting” is used because finger prints, like DNA, vary from person to person and no two people have the same DNA or fingerprints. So, when scientists identify a person’s DNA, it can only be linked back to one individual and can help to obtain information about that specific individual. DNA fingerprinting can be done through many different ways. Some ways are through blood samples from a vein or heel, saliva samples, skin samples, or hair samples. The purpose of DNA fingerprinting is to help link family members together, find things that cause diseases, to help aid in crime scene investigations, and to identify the deceased. The Pros...
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...treatment on their disorder. Vaccines and drugs are costly to produces and to administer to the affected individuals so more cost-effective therapies are required. There have been tests on gene transfer but these tests show no results to form a stable version of factor VIII or IX. More testing and clinical trials are being performed to produce a more stable version of the coagulation factors. Bioengineering coagulation factors show positive signs for victims with hemophilia which will further preclinical studies. BACKGROUND/INTRODUCTION Figure 2: Dr. John Conrad Otto Figure 2: Dr. John Conrad Otto The purpose of researching gene therapy and alternative cures for hemophilia is to cut down cost of treatment and prolong the effects of the therapy. Hemophilia dates back to ancient times. The Talmud and Arab physicians had stories about males dying from minor cuts. Hemophilia was finally recognized as a hereditary disorder in 1803 by Dr. John Conrad Otto. He saw a trend in families that female were carriers of the disorder and that mostly males were affected. Hemophilia is a genetic disorder that makes it much harder and longer for your blood to clot when you are injured. In severe cases continuous bleeding could occur after minor cuts. There are two types of hemophilia, hemophilia A and hemophilia B. Mutations in the F8 and F9 gene that causes a reduction in the coagulation factors VIII and XI. These coagulation factors are essential in the blood clotting process, also...
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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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...Summary Magnetic nanoparticles have allowed for many breakthroughs in cancer therapy. They are currently being implemented with cancer diagnostics, which allows healthcare professionals to image where cancerous areas reside in the body. For example, magnetic nanoparticle have been utilized in conjunction with MRI scans to greatly increase the image contrast, leading to the detection of small and otherwise undetectable prostate cancer metastases. Additionally, nanoparticles are being used as a physical means of destroying tumorous tissue by the use of Magnetic Nanoparticle Hyperthermia. This technology uses the power of magnets to oscillate iron oxide magnetic nanoparticles to create heat in the form of kinetic energy and has shown to drastically reduce the size of brain tumors when compared to patients who have no treatment at all. Beyond their use as diagnostics and physical tools to destroy cancerous tumors, magnetic nanoparticles are also used as a delivery agent for gene therapy. For example, magnetic nanoparticles have shown to increase the half life of the various biological matter such as DNA and RNA so that it can reach the intended target before the body’s immune response destroys the magnetic gene delivery system. In the future, research will be focused on pharmacokinetics of the nanoparticles, ensuring the nanoparticles can stay in the body for longer periods of time and that the body reacts in a positive manner to the magnetic nanoparticles. Introduction ...
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...Kimmelman Jonathan, Baylois Francois, Grass Kathleen, (2006) “Stem cell trials: Lessons from gene transfer research”, 23-26. When I first heard of stem cell research I wondered how the government could refuse Stem cell research, something that holds the cure for diseases like Alzheimer’s , Cancer or even HIV/AIDS. I didn’t know the background story and how stem cell research borders on the morality of what is ethical and what isn’t. Is it acceptable to save one life but kill another? The critics and the religious minded who believe life begins at conception see this as murder, while proponents praise it as innovative and groundbreaking, in helping save millions of lives. This Cutting-edge new experiment challenges and goes against numerous religions that oppose killing human life, which makes it so controversial. The issue of stem cell research largely involves the destruction of five to seven year old embryos, and raises the morality of this experiment. It questions our principals and makes us wonder are we really sacrificing a life or saving one? You have to choose between two wrong things. While there is immense excitement among scientists and people of the possibility of the millions of human lives stem cells can save there is also a great safety concern as well. Stem cell research has always been a very relevant topic. Over the last decade politicians, Scientists and theorists have debated on the medical advancement of stem cell research, its regenerative medicine...
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...organisms or derivatives thereof, to make or modify products or processes for specific use" (UN Convention on Biological Diversity).[1] Depending on the tools and applications, it often overlaps with the (related) fields of bioengineering and biomedical engineering. For thousands of years, humankind has used biotechnology in agriculture, food production and medicine.[2] The term itself is largely believed to have been coined in 1919 by Hungarian engineer Karl Ereky. In the late 20th and early 21st century, biotechnology has expanded to include new and diverse sciences such as genomics, recombinant gene technologies, applied immunology, and development of pharmaceutical therapies and diagnostic tests.[3] Definitions of biotechnology The concept of 'biotech' or 'biotechnology' encompasses a wide range of procedures (and history) for modifying living organisms according to human purposes — going back to domestication of animals, cultivation of plants, and "improvements" to these through breeding programs that employ artificial selection and hybridization. Modern usage also includes genetic engineering as well as cell and tissue culture technologies. Biotechnology is defined by the American Chemical Society as the application of biological organisms, systems, or processes by various industries to learning about the science of life and the improvement of the value of materials and organisms such as pharmaceuticals, crops, and livestock.[4] In other words, biotechnology can be defined...
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...Multiple Myeloma Multiple Myeloma is a form of cancer which affects the plasma cells of the body, which are white blood cells. Multiple Myeloma, first described in 1848, is a disease “characterized by a proliferation of malignant plasma cells and a subsequent overabundance of monoclonal paraprotein.” To understand how Multiple Myeloma affects an infected person’s plasma cells, it helps to have a general understanding of how normal blood cells are formed and how they act. Most blood cells develop from stem cells, which can be found in bone marrow (soft material inside our bones – the “filling”). Stem cells mature into white blood cells, red blood cells, or platelets.2 The purpose of white blood cells is to fight off infection, while red blood cells carry oxygen and platelets aid in blood clotting, which controls bleeding. Plasma cells make antibodies, which are parts of the immune system and help the body protect itself from germs and other harmful substances (Exhibit 1).2 Myeloma begins when a plasma cell begins dividing uncontrollably, which sets off a chain reaction of abnormal cell divisions. These abnormal plasma cells are called myeloma cells. Eventually, there is a large buildup of myeloma cells in the bone marrow, potentially damaging the outer, solid part of the bone. The reason the disease is called multiple myeloma because it usually affects several bones in any given infected person. The myeloma cells, instead of creating the normal antibodies, create...
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...Genetic engineering is the process of genetically modifying an organism, it’s a possibility in the near future. This process allows scientists to manipulate the characteristics of their test subjects, and if need be heal them. Human genetic engineering is genetic engineering that is conducted humans. Many people support human genetic engineering because of its potential medical purposes. However, the topic of designer babies has raised some heads, as some are against it because they believe it’s playing God. Human Genetic engineering has been viewed as a way to cure hereditary diseases. There are over 3,000 diseases as a result of a genetic disorder. Conditions such as sickle-cell anemia, Tay-Sachs disease, Duchenne muscular dystrophy, Huntington's...
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...DNA Technology BIO/240 May 6, 2013 DNA Technology INTRO – LEE DNA Technology: Cloning, Gene Therapy, and Stem Cell Research DNA technology encompasses a wide variety of applications and because of the duplicating nature of DNA, it is easy to see how humans could benefit from its manipulation. One such technology is cloning. Cloning technology comes in three forms: recombinant DNA cloning, reproductive cloning, and therapeutic cloning. Cloning Recombinant DNA cloning consists of transferring DNA fragments from an organism to a self-replicating element, like a bacterial plasmid. The fragments join with the cloning vector and are reproduced with the host cell. This technology is most commonly known for its use in genetically modified foods. DNA fragments that code for better tasting, higher nutrient qualities are spliced into regular plants to produce super foods (US Dept of Energy Genome Program, 2009). Reproductive cloning takes all the genetic information out of a cell and replaces it with DNA from the desired organism. With luck, this cell will begin to divide until it becomes an embryo and can be implanted into a host mother (US Dept of Energy Genome Program, 2009). Gene Therapy and Stem Cell Research Therapeutic cloning is by far the most controversial. This type of cloning produces human embryos for use in research, and usually for the stem cells that can be harvested from these embryos. Stem cells can be used to clone organs and body parts from the...
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...testing especially when it comes to healthcare and when it leads to vanity. Genetic testing examines a person's genetic code by using a sample of blood or other body fluids/tissues, for health or medical identification purposes; it’s also a type of medical test that identifies changes in chromosomes, genes, or proteins (Genetic Testing). The Human Genome Project, human cloning and stem cell research are all part of this group. Genetic cloning is the reproduction of a new organism that is, at all stages of development, genetically virtually identical to a currently existing, or previously existing, being (Clone). Stem cell is an unspecialized cell that gives rise to a specific specialized cell, such as a blood cell, and they have the ability to divide for indefinite periods in culture and to give rise to specialized cells (Stem Cell). The Human Genome Project was an international research effort to determine the sequence of the human genome and identify the genes that it contains. The Human Genome Project formally began in 1990 and was completed in 2003 (Human Genome Project). “The purpose of the Human Genome Project has allowed researchers to begin to understand the blueprint for building a person. As researchers learned more about the functions of genes and proteins, their knowledge has had a major impact in the fields of medicine, biotechnology, and the life sciences” (What Was the Human Genome Project and Why...
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...paper will give a description of the sickness through the discussion of the causes, symptoms, and possible cures. Sickle Cell Disease (SCD) is a "group of inherited red blood cell disorders."(1) These disorders can have various afflictions, such as pain, damage and a low blood count--Sickle Cell Anemia. Sickle cell disease is the most common inherited blood disorder in the United States. Approximately 80,000 Americans have the disease. In the United States, sickle cell disease is most prevalent among African Americans. About one in 12 African Americans and about one in 100 Hispanic Americans carry the sickle cell trait, which means they are carriers of the disease. Sickle cell disease is caused by a mutation in the hemoglobin-Beta gene found on chromosome 11. Hemoglobin transports oxygen from the lungs to other parts of the body. Red blood cells with normal hemoglobin (hemoglobin-A) are smooth and round and glide through blood vessels. In people with sickle cell disease, abnormal hemoglobin molecules - hemoglobin S - stick to one another and form long, rod-like structures. These structures cause red blood cells to become stiff, assuming a sickle shape. Their shape causes these red blood cells to pile up, causing blockages and damaging vital organs and tissue. Sickle cells are destroyed rapidly in the bodies of people with the disease, causing anemia. This anemia is what gives the disease its commonly known name - sickle cell anemia. The sickle cells also block...
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