Somatic gene therapy is an approach to the treatment of a variety of human inherited and acquired disorders. The major goals of gene transfer technology that could lead to successful gene therapy are techniques that lead to (1) high level and efficient transfer of genes into appropriate target cells, and (2) high-level and appropriate expression of these transferred genes. Safe and efficient transfer of the bacterial neo® gene using retroviruses has been accomplished using appropriate cell lines to package viruses (packaging lines). Although a high level of success has been achieved in the expression of genes in tissue culture cells, high levels of expression have not been achieved to date using gene transfer into the cells of intact animals. Thus, new retroviral vectors leading to high-level expression of the foreign genes they contain are the major goal of current experimentation.
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