ABSTRACT

Recent advances in molecular biology and the human genome project have brought about improved understanding of the genetic basis for human disease. The wealth of knowledge about genetic causes of human disease provides compelling and substantive support for the development of gene therapy. Today, many incurable diseases such as inherited genetic diseases, cancer, and AIDS are under consideration as candidates for gene therapy. In some

cases, gene therapy represents the only long-term hope of treatment for these diseases. After a decade of extensive research on gene therapy, it is now clear that the vehicle with which the therapeutic gene is introduced into the target cells of the body is the key to the success of an effective cure.