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Gene therapy is reinserting certain genes that helps deal with genetic diseases. There are three basic forms of this gene therapy. The first is Gene Inactivation Therapy in which the transferred gene neutralizes the proteins and evens out the amount or rids of the defective proteins. Another type is Gene Augmentation Therapy where the original form of the gene or the normal form of the gene is inserted into one of the cell’s chromosomes. This procedure is used normally when a gene with little activity or a deleted gene is the cause for the genetic disease. The third type of gene therapy is Gene Replacement Therapy. This form is used when the genetic disease involved specific genes that are necessary for proper functioning. The normal gene being put in place of the mutant gene accomplishes this form of gene therapy.
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.
There have been four somewhat recent successful gene therapy treatments. The four deal with correcting hemophilia, bone marrow transplants, skin cancer, and vessel growth. In the success with the bone marrow transplants, French researchers collected bone marrow cells from patients, used gene therapy to correct the bone marrow, and then returned the bone marrow to the patient. This was 80% successful as reports 16 months after the transplants showed. Squamous cell carcinoma, skin cancer of the head and neck, was treated using gene therapy as well. The fourth trial was where DNA was used to carry a substance that stimulates blood vessel growth to damaged heart tissue and in this trial there was much success noted.
The beginning of Gene Therapy began in the late 1980’s, which was completely unsuccessful. In the fall of 1999 the death of University of Pennsylvania trial participant, Jesse Gelsinger was followed by much public outcry and legal problems that put an immediate halt on all gene therapy research. The reason Jesse was being treated with Gene therapy was to attempt to cure the teenager’s rare liver disease.
Over 20 years after the proclamation of these specific ethical guidelines, we are introduced to the University of Pennsylvania’s Institute for Human Gene Therapy’s study on a delivery mechanism for gene therapy that resulted in the death of an 18 year old research subject Jesse Gelsinger. Gelsinger suffered from partial OTC (ornithine transcarbamylase) deficiency caused by a defective single gene (Obasogie, 2009).
Gene therapy is the application of the technique where the defect-causing "bad" genes are replaced by correct "good" genes. The idea of gene therapy is to treat the disease by correcting the "bad" DNA (Deoxyribonucleic acid) rather than the current me thod of providing drugs, or proteins not produced by the defective gene. Gene therapy addresses the problem first hand by directly working with the genetic information causing the disease. From the book Shaping Genes, Dr. Darryl Macer says "It is like f ixing a hole in the bucket, rather than trying to mop up the leaking water." There are two kinds of gene therapy, somatic cell gene therapy and germline gene therapy.
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.
Human gene therapy is a method used in the medical field that treats diseases at a molecular level, by solving the source of the problem; our genes. Today, diseases and disorders are commonly treated by solving the symptoms, the surface of the problem. Many disorders and diseases are caused by defective proteins and within those defective proteins are damaged and defective genes. These defective genes can be treated through gene therapy. Gene therapy is not new and has been developed and improved by researchers for the past couple years. Being an experimental technique, gene therapy also has its pros and cons, but so far is showing positive and rising success rates.
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
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.
Genetic engineering is now being used to create new medicines and therapies for many disorders and diseases, and also to improve agricultural plants and animals to produce bigger yields or enhanced nutrient composition and food quality. In Gene therapy, copies of healthy human genes produced in bacteria can be inserted into human cells with defective or missing genes, to fix the problem. Gene therapy is promising because it can use to treat genetic
Gene therapy focuses on the replacement of defective genes with modified functioning genes. Many diseases are caused by a defective gene meaning the body is incapable of producing essential proteins or enzymes. In its simplest form, gene therapy aims to identify the defective gene and fix this gene with the replacement of a normal gene (Senn).
Viral vectors use viruses to transport a modified gene into a patient's body. They are right now be...
Current research methods of transfection, delivering foreign DNA into cells, have capitalized on using non-viral vectors because of the recent advantages researchers have been able to exploit. The process of transfecting cells runs into a number of problems by way of the cell’s own defense mechanisms. Vectors must be able to not only enter the cell past the cell’s membrane but also must be able to make its way into the cell’s nucleus to access the targeted genetic material. The problem with traditional transfection methods is that they are not able to enter the cell in high efficiency without triggering an immune response. This, coupled with the inability for prolonged gene expression in vivo even once transfected, results in a very expensive and ineffective method for introducing a foreign plasmid into the cell. In the past viral vectors had been used with a degree of success in vitro, but because they lack a high degree transfection efficiency and duration of gene expression using them for transfection could not produce substantial practical applications. Another problem is that these laboratory-engineered viruses had low success rates in vivo due to activating an immune response. New techniques are being discovered by modifying non-viral vectors in novel ways, producing increasingly effective methods for transporting DNA into cells with the hope of clinical application and advancing gene therapy.
Instead, a carrier called a vector is genetically engineered to deliver the gene. Certain viruses are often used as vectors because they can deliver the new gene by infecting the cell. The viruses are modified so they can’t cause disease and will not be fought off by the patient’s immune system. The vector can be injected or given by IV directly into a specific place in the patient’s body. The vector can also be introduced after a sample of the patient’s cells are removed and exposed to the vector in a lab.
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.
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.
Genes are made of DNA – the code of life (Gene Therapy- The Great Debate!). The changes in genes may cause serious problems, which we called genetic disorder. In theory, the only method to cure genetic disorders is gene therapy, which basically means the replacement of genes in order to correct the loss or change in people’s DNA. Although gene therapy gives patients with genetic disorders a permanent cure, it is controversial because it has safety and efficacy problems, and raises ethical issues.
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.