Jesse Gelsinger The objectives for this ambitious effort are to learn more about heredity of disease and to discover the genes that would aid in gene therapy. Advances in gene therapy strive to treat hereditary Importance and Uses of Gene Therapy for Human | Essay
What is gene therapy? | EssayBiz
Searching for writers Place an order. Track the Progress. Receive a Paper. All revisions are for FREE. For over fifteen years, genes have been used to produce, with biotechnological techniques, gene therapy essay, pure proteins that are used as biopharmaceuticals insulin, growth hormone, blood coagulation factors, erythropoietin, etc. Gene therapy is a medical technology in which DNA is directly used as a pharmaceutical substance, gene therapy essay. With this technique, gene therapy essay, the genes or fragments of these are inserted into the human body to prevent, treat, or treating a disease.
The greatest difficulty encountered in transferring the gene is achieving a sufficient level of efficiency. Gene therapy can potentially cure many diseases, both genetic and acquired. The experimental clinical gene therapy began in To date it around involving nearly 3, patients were made. Some of these trials have been conducted or are currently underway in Switzerland. However, the results obtained so far are strictly experimental. The term "somatic" is opposed to the term "germinal. Currently, it is impossible to completely control the genetic alterations that are brought about by a gene transfer. Consequently, there is the risk of provoking physiological anomalies that would be transmitted to subsequent generations; furthermore, the alteration of heritable genetic material raises a series of ethical and moral questions.
For these reasons, gene therapy essay, the genetic modification of germ cell lines is not considered an adaptable strategy, and its clinical trial is prohibited in Switzerland as in almost all other countries in the world. With gene transfer on somatic cell lines, gene therapy essay, the genome of tissues muscles, lungs, brain, bones, kidneys, heart, etc. is changed, without this modification being passed on to the next generation; the genetic alteration concerns only the patient on whom it was made. This does not mean that such therapy is risk-free. We are studying the complications of random insertion of a foreign gene into the genome; according to the carrier that is used, the risks can be more or less high, thus determining the indications and contra-indications of a given treatment.
Lungs, brain, bones, kidneys, heart, etc. Theoretically, all diseases can be treated through gene expression gene therapy essay. Commonly, gene therapy is thought to help heal hereditary diseases such as:. In reality, this therapy can also treat diseases that affect those genetically predisposed, but which depend heavily on environmental factors, such as:. In vivo, physical methods have limited applicability to the transfer of genes to surface tissues, while they are more conveniently used for ex vivo transfer. Direct injection into the tissues is effective only when gene therapy essay does not gene therapy essay a large amount of expression of the transferred gene for example, when patients are treated with expression vectors for VEGF, growth factor of vascular endothelium.
The advantage of chemical methods is the ability to build the desired vector by combining the different chemical compounds, which are easily obtainable; however, the efficiency is to 1, times lower than that of biological vectors. Future improvements of this method, such as the creation of compounds formed by chemical and biological substances, could lead to the formation of "virosomes" or "artificial viruses. Gene therapy essay fact, these vectors can be designed to accumulate in the desired body compartments, due to surface molecules that are specific ligands. In biochemical methods, DNA is conjugated with proteins that can enter cells by endocytosis, gene therapy essay, for example, through internalization mediated by a receptor.
These methods offer the advantage of specifically targeting certain cells, and such specificity is not possible with chemical methods. Most of these viruses are constructed in such a gene therapy essay that they can only replicate in genetically manipulated cells. The major advantage these carriers offer is that they can transport genes with very high efficiency. Some vectors for example, lentivirus allow the integration of the introduced gene into the host cell genome. In this way, the expression of the introduced gene is permanent. The main risk we face is that the virus gives rise to particles capable of reproducing in an uncontrolled way.
However, the use of modern protocols appears to have significantly reduced this risk. Some vectors for example, recombinant adenoviruses transport toxic components such as capsid proteins into the cell; this may preclude their use for non-serious diseases. Finally, vectors such as AAV do not allow the packaging of genes with a long DNA sequence, causing a severe limitation for the treatment of diseases such as muscular dystrophy and cystic fibrosis, where the gene of interest is larger than the space available in vector. Many studies and experiments are conducted to make recombinant viruses capable of attaching to specific surfaces within the body to allow a systemic tissue-specific gene transfer. They are causing a serious limitation for the treatment of diseases such as muscular dystrophy and cystic fibrosis, where the gene of interest is larger than the space available in the vector.
Currently, many studies and experiments are conducted with the aim of making recombinant viruses capable of attaching to specific surfaces within the body, so as to allow a systemic tissue-specific gene transfer. Currently, many studies and experiments are conducted with the aim of making recombinant viruses capable of attaching to specific surfaces within the bodyto allow a systemic tissue-specific gene transfer. search order now. Need a custom essay? Login with Facebook. What is gene therapy? What does 'somatic' gene therapy mean? What diseases can be treated with gene therapy? Commonly, gene therapy is thought to help heal hereditary diseases such as: - muscular dystrophy - cystic fibrosis - hemophilia - type I diabetes - metabolic diseases phenylketonuria - Physiological anomalies mucopolysaccharidosis, Gaucher syndrome, etc.
In reality, this therapy can also treat diseases that affect those genetically predisposed, but which depend heavily on environmental factors, such as: - cancer - cardiovascular diseases - Neurodegenerative diseases Alzheimer's disease, Parkinson's disease, etc. Finally, gene therapy can also treat acquired diseases such as: - Various types of trauma bone fractures, wounds, burns - Ischemias tissue necrosis caused by an interruption of blood supply - Infections Which gene transfer vectors are used today? What is "in vivo" and "ex vivo? Physical methods - hydrodynamic pressure. We will write it for you. Order now. All you need to know about Neuroendocrinology. All you need to know about Big data management. All you need to know about digital special effects.
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Gene Therapy
, time: 5:45The objectives for this ambitious effort are to learn more about heredity of disease and to discover the genes that would aid in gene therapy. Advances in gene therapy strive to treat hereditary Jesse Gelsinger Gene therapy is a medical technology in which DNA is directly used as a pharmaceutical substance. With this technique, the genes or fragments of these are inserted into the human
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