ABSTRACT
The concept of gene therapy for human disease has become very popular during the past decade or so. The potential for genetic approaches to therapy has been recognized and strengthened not only by traditional funding mechanisms but also by a number of timely and targeted funding opportunities from agencies such as the National Institutes of Health and from disease foundations such as the Cystic Fibrosis Foundation and others. Currently available tools for gene transfer into human cells and tissues, including viral and nonviral reagents, are largely inadequate for the difficult job of transferring a therapeutic gene efficiently and safely into defective human cells in a tissue-specific way to ensure effective genetic modification of only specific target cells and an effective attack on a specific genetic function. A number of additional viral vector systems are becoming available for therapeutic gene transfer in a variety of disease models, including disorders of the lung. Gene therapy is in its earliest stages of development.
