ABSTRACT
It is important to realize that targets for therapeutic genes will differ. In certain cases the gene must be delivered to a specific cell type, for example replacement of globin genes in inherited hemoglobinopathies in which the gene must be delivered to erythroid cells. In other cases it is only necessary to increase the level of a specific protein, for example a hormone, in the body. Here any cell type that will allow the product to be secreted into the blood system will be a suitable target.
