Gene Therapy Essay

1392 words - 6 pages

Science Research TaskEthics of GeneticsAydhen Roopra1.a) I have chosen to research gene therapy.b) Issues within society that gene therapy addresses are genetic disorders or mutations, these can include cancers, diabetes and Gaucher disease. Gaucher disease is a genetic disease in which a thick substance accumulates in certain cells and certain organs, possibly causing symptoms such as bruising, fatigue and the enlargement of the liver and spleen. Gaucher disease is hereditary, caused by the deficiency of the enzyme glucocerebrosidase. This enzyme is responsible for the accumulation of the glycolipid glucocerebroside. When the enzyme is deficient, the glycolipid can accumulate abnormally, particularly in white blood cells, in the spleen, liver, kidneys, lungs, brain and bone marrow.c) Gene therapy is being used to cure Gaucher disease by injecting a retroviral vector with the spleen focus-forming virus promoter driving the enzyme glucocerebrosidase. This method has been used on mice carrying the Gauchers disease. The aim of this study was to generate mice with a symptomatic form of Gauchers disease for use in the development of cell and gene therapy in the disorder. The results of the study concluded that gene therapy is indeed a feasible method for treating patients with Gauchers disease, as long as there is full or partial ablation of bone marrow, otherwise it is not likely to be an effective method."Gene Therapy Can Correct the Phenotype of GD Mice.The GD mice were used in proof of principle experiments to investigate the feasibility of gene therapy for type 1 GD. A retroviral vector with the spleen focus-forming virus promoter driving GCase expression (GC vector) was used. The control vector (GFP vector) was identical to the GC vector with the exception of the GCase gene."(Extract from The Proceedings of the National Academy of Sciences of the United States, 2006)[Gene Therapy Can Correct the Phenotype of GD Mice, The Proceedings of the National Academy of Sciences of the United States, 2006][GD - Gauchers DiseaseGCase - glucosylceramidase (gene)]2.Gene therapy is not a new field; it has been around for decades, yet gene therapy still has very limited success because it is very hard to introduce new genes into cells of the body and keep them working. Gene therapy, for the use of treatment in Gauchers disease, is a progressing method. It is effective in that, for Gauchers disease, it is a valid method of treatment, yet it requires additional surgical procedures in order for a likely success for the case of human treatment. Gene therapy is essentially dependant on the development of the gene delivery vector. Physical techniques have been introduced to improve the effectiveness of gene transfection such as electroporation, ultrasound and hydrodynamic pressure.In some diseases, gene therapy is successful; when a normal gene is introduced to a large number of cells, generally in the millions, depending on the severity of the disease, when once all...

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