Gene therapy involves the expression of a gene or genes within a patients specific target cells, which change the functional properties of the cell such that a therapeutic benefit is produced for a patient. For example, in a person with sickle cell disease, the adult globin gene has a single base pair mutation. This changes the structure and function of the globin protein producing a severely debilitating disease in homozygous individuals. The goal of gene therapy for this disease is to replace the defective gene in the patients blood cells by a normal gene so that the normal hemoglobin protein can be produced.
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