The emergence of technically sophisticated gene transfer approaches for a diverse array of inherited and acquired human diseases has required preclinical assessment in animal models using conditions and reagents similar to those that would be used in the clinical setting. Murine models have contributed significantly to the development of gene therapy. In this context, this chapter reviews the basic concepts of gene therapy, the gene transfer vectors, and the uses of gene transfer in murine models. Gene therapy can be loosely defined as the transfer of genetic material into a cell, tissue, or organ for treating and/or preventing disease. Delivery can be local or systemic, depending on the application. Gene transfer can also be used to transfer regulatory sequences, but the primary application of gene transfer is to deliver proteins to be used within the target cells, in their local milieu, or to be secreted for systemic use. Other uses of gene transfer, such as the delivery of antigens for vaccination against the infectious agent from which the antigen is derived, are variations on the basic gene therapy theme. Gene transfer can also be used to develop animal models.
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