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Effects of gene therapy
Various genetic disorders
Various genetic disorders
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Gene therapy methods
Gene therapy is one of the most rapidly growing techniques in the medical field. One out of ten people are affected by genetic disorders. Defective genes that code for an incorrectly formed protein, resulting in a severely hindered function, cause genetic disorders or process that are usually lethal. The essential idea was to replace the defective genes causing the disorder by introducing a confirmed healthy form into the patient through some sort of vector. Vectors are fragmented down into two groups, viral and non-viral. Viral vectors have been prosperous in some gene therapy applications, but there are possibilities that the patient’s immune system may defend against the virus and render the application insufficient. These vectors have patient’s immune system defend against the virus and render the application unusable. It also, has been successful in treating patients with severe combined immunodeficiency (SCID), in part due to the weakened immune system of the patients. Methods that are used in the following papers utilized viral vectors to introduce the desired genes into the patients. Severe combined immunodeficiency that is linked to the
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have developed a new viral vector to deliver the cystic fibrosis. PIV vectors was made through the use of recombinant human PIV3 encoding GFP, CFTR, and F508CFTR cDNA as separate genes generated from the cDNA antigenome of full-length hPIV3 JS strain. After retrieval, PIV replicated in HEp2 cells to a titer comparable to the JS wild-type strain suggesting that GFP, CFTR, and F508CFTR did not adversely affect growth capacity of PIV in producer epithelial cell lines (Zhang et al., 2009). In vitro model of human airway epithelium was used to test the effectiveness of parainfluenza virus with the CFTR gene (Zhang et al., 2009). There was a success in delivery of the CFTR gene to the surface cells (Zhang et al.,
Cystic Fibrosis (CF) Pathophysiology: Cystic fibrosis is a genetic disease of the secretory glands that affects the respiratory and digestive system. It mainly affects the lungs, pancreas, liver, intestines, sinuses, and reproductive organs. Cystic fibrosis affects the cells that produce sweat, mucus, and digestive fluids. Mucus becomes thick and sticky, causing build-up in the lungs and blocking airways, making it easier for bacteria to develop. This prompts repeated lung infections and can cause severe lung damage after some time.
Modern molecular genetics has given hopes and heartaches to thousands of people around the world. These people are looking towards gene therapy for an answer to their questions. To some people such as NIH director Harold Varmus the answer is a better understanding of basic genetic research and to others the answer is a cure, a hope, that their lethal disease will someday be cured. This essay touches on the background of gene therapy for Cystic Fibrosis (CF), current social and ethical issues facing gene therapy for CF, and some thoughts on the importance of this controversial subject.
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.
Either transduction or transfection can be used to get the therapeutic genes into the patients system. Transfection is when the genes are introduced physically or chemically in a way that allows the cell membrane to be temporarily permeable to a foreign DNA. In the second method used for gene therapy, transduction, there is a beneficial gene added into the genetic material of the virus, which then is allowed to infect the target cell which is the indirect transfer method for gene therapy.
Those who have relatives diagnosed with Cystic Fibrosis are at an automatic increased risk to have the disease. For example, in order for a child to have cystic fibrosis, their parents must be carriers of the CF gene. “One CF gene fr...
Cystic Fibrosis is an inherited disease characterized by the buildup of thick, sticky mucous that can cause severe damage to the body’s organs. Mucous is usually a slippery substance that lubricates and protects the linings of the airway, digestive system, reproductive system and other organs and tissue. Problems with digestion can lead to diarrhea, malnutrition, poor growth, and weight-loss. Due to the abnormally thick mucous it can can clog airways, leading to breathing problems and bacterial infections in the lungs. Bacterial infections can lead to coughing, wheezing and inflammation. Overtime these infections can lead to permanent damage in the lungs including the formation of scar tissue, known as fibrosis and cysts in the lungs (Genetics Home Reference, 2013). The symptoms and signs of this disease vary but mostly include progressive damage to the respiratory system and chronic digestive system problems. An individuals’ lungs who are infected by cystic fibrosis have bacteria from an early stage. This bacteria can spread to the small airways, leading to the formation of bacterial micro-environments known as biofilms. Biofilms are difficult for antibodies to penetrate, therefore the bacteria repeatedly damage the lung and gradually remodel the airways, resulting in difficultly to eradicate the infection (Welsh, 1995). Cystic fibrosis patients may even have their airways chronically colonized be filamentous fungi and/or yeasts. Most men with cystic fibrosis have congenital bilateral absence of the vas deferens (CBAVD), a condition in which the tubes that carry sperm are blocked by mucous and do not develop properly. As well, women may experience complications in pregnancy. Either the c...
Gene therapy is an experimental technique that allows doctors to insert a gene into a patient’s cell rather than using drugs or surgery. Gene therapy is a process of which defective or undesired genes in the body with “normal” genes. A vector is re-engineered to deliver the gene to a target cell. Then the gene is transferred to the cell’s nucleus and must be activated in order to function. The main focus of gene therapy is to replace a lost or improper gene with a new functional copy into a vector that is inserted into the subject’s genome by way of penetrating its DNA. Gene therapy can be done outside of the body known as ex vivo by way of taking cells form patients bone marrow or blood and then growing them in a laboratory. Thus the corrected copy of the gene is inserted into the cells before being put back into the body. Gene therapy can also be done in vivo which can be done directly to the patient’s body. The word gene therapy really defines the management of genetic information that is encased in the cells, however, in most recent procedures the available technology is closely related in adding new genetic information, and many researchers favor the term gene transfer rather than gene therapy to mirror the reason that the purpose of gene work cannot always be therapeutic.
Gene therapy works by introducing new and functioning genetic material to damaged genes to help it function and to produce beneficial proteins. If a gene is inserted directly into a cell, it usually will not function. So to complete this task, a vector, a modified virus is used to carry and deliver the new gene. There are two different categories of vectors than can be utilized in this process; recomb...
Gene therapy gives people who suffer from genetic diseases a chance to lead a normal life. Dangerous diseases, such as AIDS, SCID, Thalassemia and ADA can be cured successfully. In September 5, 2006, two people with advanced melanoma received Gene therapy and they got recovery soon. This is a breakthrough in cancer gene therapy. Gene therapy uses patients own cells to cure diseases, and, therefore, no rejection to their bodies. Furthermore, patients could get permanent cure from gene therapy without recurrence.
Gene therapy is a provisional technique that is the insertion of normal genes into the cells where there is a missing or miscoded gene to fix a genetic disorder. In the 1960s and early 1970s,
The types of diseases that can be treated by gene therapy are those where replacement of a single gene is able to cure the disorder. Some of the main diseases currently being researched in gene therapy studies include cancer, hemophilia, sickle cell anemia, muscular dystro...
Viral vectors use viruses to transport a modified gene into a patient's body. They are right now be...
[Cover: discussion about how risks are balanced during risk assessment, why this is a difficult task -> proposing a set of principles and practical measures that might assist both researchers and patients, to enable more informed decisions about risk]
Gene therapy enables patients to survive incurable diseases. In the field of genetic diseases, ADA-SCID, CGD and hemophilia are three main ones. ADA-SCID is known as the bubble boy disease. CGD is related to immune system that would lead to fungal infections which are fatal. Patients with Hemophilia are not able to induce bold bleeding (Gene therapy for diseases, 2011). Gene therapy also has good effects on cancer treatment and neurodegenerative diseases, which include Parkinson’s disease and Huntington’s disease. Viral infections, including influenza, HIV and hepatitis can also be treats by it (Gene therapy for diseases, 2011). According to the Science Daily in 2011, gene therapy now can apply to heart failures and neurologic diseases as well.
Position Paper: Gene Therapy in Humans. Advancements in science and medicine are usually accompanied by 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 scientific world and everyday life. New technology, discoveries, and breakthroughs are rapidly occurring in the field every day.