ABSTRACT

Gene therapy, the transfer of genes into cells for a therapeutic effect, is a new approach to the treatment of disease. The potential applications of gene therapy are far reaching since there are over 4000 known human genetic diseases and virtually every human disease is profoundly influenced by genetic factors. Gene therapy, the transfer of genes into cells for a therapeutic effect, is a new approach to the treatment of disease. The potential applications of gene therapy are far reaching since there are over 4000 known human genetic diseases virtually every human disease is profoundly influenced by genetic factors. Gene delivery systems can be classified as either viral or nonviral. For viral gene transfer, one of several different types of viruses is engineered to deliver genes. Non-viral gene transfer systems are based on a variety of technologies that employ physical/chemical means to deliver genes. Retroviruses used for gene transfer are derived from wild-type murine retroviruses.