What is Gene Therapy?
Gene therapy is a relatively new concept, it is an experimental technique that uses genes to theoretically treat and prevent various types of Illnesses/Genetic Disorders such as Haemophilia, Cancer, Chronic Granulomatous Disorder and Cystic Fibrosis. The process of gene therapy involves the use of a Viral Vector (Most Common Form) other forms of delivery which are non-viral include the injection of naked DNA, sonoporation and magnetofection.
Figure 1 .1 Shows us how an unmodified Virus under normal conditions work.
How It Works?
The viruses DNA is removed and replaced by the needed DNA that corresponds to the specific required gene, which is then to be inserted into a human body, once its inside, the cell's gene-reading machinery uses the information in the gene to build RNA and protein molecules, these proteins can therefore carry out their specific jobs within the cell(s).
Figure 1.2 Shows How to Create a Viral Vector.
Another Type of Gene Therapy:
Germ line gene therapy involves the modification of germ cells (gametes) that will pass the change on to the next generation. With germ line therapy genes could be corrected in the egg or the sperm that is being used to conceive. The child that results would be spared certain genetic problems that might otherwise have occurred. Because every cell descends from the fertilized egg, every cell in the offspring would possess the transplanted gene. This would be a far more effective way of transferring genes than the ones presently used in somatic cell therapies, where genes into the cells of children or adults usually enter only a small portion of the person’s cells and eventually stop functioning.
Gene Therapy sounds very appealing, a quick fix that could s...
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...m line gene therapy are not yet born they can’t have the decision to choose whether or not they want to undergo the treatment
Figure 1.5 poses the question, Gene Therapy will it make us intellectual property and products?
Conclusion:
Once gene therapy develops enough to enter mainstream treatment is will be quite expensive and not everyone will be able to become disease free instantly, but with time this price will go down. Gene therapy is a possible permanent solution to all sicknesses and diseases no matter how severe they are, allowing everyone no matter what to live a normal life, the disadvantages are there but only because our knowledge of the treatment is still fairly limited once that changes, gene therapy will definitely be a viable solution to all our medical/health problems we face today such as mentioned Cystic Fibrosis, Choroideremia and Diabetes.
Germline gene therapy is where the correct "good" gene is inserted into the germline in place of the defective "bad" gene, and when reproduction occurs the gene will be passed on to the progeny. Inserting the "good" gene into the very early embryo sta ges of development allows for both germline and somatic cells to be corrected. Government has limited the research to only somatic cell gene therapy such as performed in Cystic Fibrosis research.
It was in the 1980’s that scientist began looking at alternative ways of treatments, one is gene therapy. Scientist would insert human genes into a bacteria cell. Then the bacteria cell would transcribe and translate the information into a protein. Once that is done the scientist would then introduce the protein into human cells. Gene therapy can be simply viewed as inserting bits of foreign DNA into a patient’s tissue in hope...
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...
Although the highly technical aspects of human gene therapy are somewhat complex, the basic concept is very straight forward. The goal of gene therapy is to correct mistakes that have occurred within the genetic material, or DNA, of the living cell. In very simple terms, DNA is often thought of as the "language" of the biological functioning of organisms. This language is organized by letters (nucleotide pairs), words (codons), sentences (genes), and books (genomes). Before being able to repair the damaged or defective genetic material, the location of the gene or genes causing the dysfunction in the individual must be determined.
Gene therapy is a new approach to treating diseases by changing a person’s genes. Not only does gene therapy hope to be used to cure genetic diseases, but there are also promises of disease prevention. There are two types of gene therapy somatic and germline. Somatic gene therapy hopes to change the gene of the patient, but this change of expression in the gene will not be passed down to the next generation.
...s of gene therapy is that the mortality rate is very high. This is because Immune system may attack cells and cells may attack vital organs. Furthermore, ethical issues should be dealt in a positive way. The technological institute has to reduce the unnecessary expenses of the treatment. I highly suggest the government investing more money on the development of gene therapy.
So Gene Therapy, being a process solely aiming to heal genetic issues in human beings, is a technology which is desirable for our modern society. If a method can be produced, which cause...
"The aim is to decrease the fear of a brave new world and to encourage people to be more proactive about their health. It [Gene therapy] will help humans become better physically and even mentally and extend human life. It is the future” (Hulbert). Dr. Hulbert, a genetic engineer, couldn’t be anymore right; more time, money, and research needs to be put into gene therapy and genetic engineering, since it can cure certain illness and diseases that are incurable with modern medicine, has fewer side-effects than conventional drugs or surgery, and allows humans to be stronger physically and mentally at birth. Gene therapy or genetic engineering is the development and application of scientific methods, procedures, and technologies that permit direct manipulation of genetic material in order to alter the hereditary traits of a cell, organism, or population (NIH). It essentially means that we can change DNA to make an organism better. Genetic engineering is used with animals and plants every day; for example with genetic...
Gene therapy is a method used by doctors that ‘uses genes as medicine’. It repairs faulty or damaged gene copies by transferring a therapeutic or working gene into specific parts of faulty gene copy. This means that gene therapy can be used to replace a faulty gene or to introduce a new gene whose function is to modify the clinical course of a condition or to cure a problem in the gene (Refer to image 2). Image 1 shows how gene therapy is completed using an adenovirus vector while image 2 explains the method of gene therapy and shows how cells are removed, altered and injected back into the patient. Gene therapy has potential to treat and cure many medical conditions such as various types of cancer, degenerative diseases and viral infections such as AIDS. Research into gene therapy still needs to be done before this approach to the treatment of certain conditions will realise its full potential. Trials are being conducted in the United States and Europe and a modest number initiated in other countries, including Australia. The majority of these trials are focussing on treating acquired conditions such as cancer (European Society of Gene & Cell therapy, 2011).
There are two types of genetic technology. Somatic gene therapy is used to repair genes that do not function properly in a person. Not that controversial because it only changes genes in organs and is not passed on to the next generation (p. 116, Garreau, 2005) .
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 has become an exciting and controversial issue on the scientific and medical horizon. Science offers new technologies that, in the future, will be able to treat and cure common genetically passed diseases. However, as it is an extremely broad subject, some time must be dedicated to its interpretation and explanation. First, a general definition of gene therapy is required. Genethics, the Clash between the New Genetics and Human Values, by David T. Suzuki and Peter Knudtson, defines gene therapy as "the medical replacement or repair of defective or faulty genes in living human cells." It is not really so elementary as the definition would imply. Within gene therapy there lie certain aspects, some more controversial than others, some more achievable and probable than others. The ethical question must be addressed at each turn. However, all of this will be discussed at greater length subsequently.
Gene therapies necessity, stems from the fact that it can cure, life threatening gene based diseases, with minimal after effects. Although there are some ethical and physical issues that can occur through this technology, when comparing it to the other genetic technologies, it’s quite reliable. Although it is in its infancy, the technology has still been present for several years, with the first case of gene therapy being done in 1990. Sure, there have been some complications, but its still a growing technology with a strong future in front of
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.
...ring deadly diseases and preventing abortions. In order for gene therapy to one day become effective much more research needs to be done to discover the consequences of altering specific genes. Also the technology of gene therapy needs to be cost effective so people who need help are able to get help. In the end gene therapy in humans needs to come a long ways before it will be widely accepted but there is great potential in the technology and it needs to be pursued.