Gene Therapy

Essay by 00280602High School, 10th grade August 2014

download word file, 6 pages 0.0

Science Research Task

Ethics of Genetics

Aydhen Roopra


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...