ABSTRACT
Gene therapy involves the introduction of DNA or ribonucleic acid (RNA) into target cells to either express or suppress the biosynthesis of proteins (1,2). The ability tomanipulate protein expression in humans could provide a cure or treatment for many diseases that are currently untreatable by conventional drug therapy. The potential therapeutic utility could be greatest for inherited diseases, such as cystic fibrosis and hemophilia, since the genetic basis of these diseases is well known. In principle, replacement of a single defective gene in the affected cells could permanently halt the symptoms. Gene therapymay
also have a unique impact on certain acquired diseases such as cancer and AIDS by virtue of the ability to use the genetics of the affected cell to mediate its own destruction (3-6). The therapeutic success of gene therapy is largely dependent on the development of efficient delivery systems for DNA.
