What Are The Risks Associated With Gene Therapy?

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What are the risks associated with gene therapy?

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This paper discus about the Gene therapy involves the introduction of genes. Viruses can usually infect more than one type of cell. Thus, when viral vectors are used to carry genes into the body, they might infect healthy cells as well as cancer cells. The vectors used in gene therapy, and genetically modified cells by these vectors are classified as genetically modified organisms. What are the risks associated with gene therapy?


Gene therapy involves the introduction of genes into individual cells and tissues to treat chronic diseases and genetic disorders in which disruption of mutant genes are replaced with functional genes. A broader definition of gene therapy includes all applications of DNA technology to treat disease. It is difficult to estimate the risks and consequences of the discrepancy between the actual behavior of the genetically modified organism and the expected behavior. Most risks are related to the production and use of vectors to deliver a foreign gene into a cell. If the target cell is already infected by a virus, a recombinant vector can transform the infectious virus. Retroviruses are chosen that do not have homologous sequences with viruses that infect humans. To prevent the spread of viral genes, limiting the use of vectors to specific venues. Another type of danger is due to the ability of retroviral vectors to induce the production of tumors. To avoid this, retroviral vectors inserted into suicide genes.

Discussion & Analyses

Despite the precautions the risk can not be eliminated completely. Another kind of risk is related to the genetic modification of germ cells. The technology opens up several avenues of investigation, as the study of the basic biology of sperm production, or use of these gametes precursor cells in experiments in genetic engineering and gene therapy, since alterations to the following generations would.

In June 1992, a team from the University of Michigan, led by James Wilson, reported treating a patient of 29 years who was born with a severe form of hypercholesterolemia caused by complete dysfunction of the LDL receptors, which are proteins found on the surface of many cells, mainly in the liver, and are responsible for transporting cholesterol within these cells. When a person has too few LDL receptors, or they do not work (as was the case with this patient), cholesterol accumulates in the blood, sticking on the walls of blood vessels and in plaques in artery walls, which eventually causes the heart attack or stroke. Even with the most complete current treatments, diet, cholesterol lowering drugs and surgery, these patients usually die of heart failure in his youth. Surgeons removed the patient 10% of his liver and researchers tissue disintegrated into its components: the liver cells. These were treated with a retrovirus (a type of virus whose genetic material RNA molecule), which had introduced a normal copy of the gene for the LDL receptor. Three days later they returned to the liver cells of the patient through a special catheter. The results were ...
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