Naked DNA gene transfer in mammalian cells.

Abstract

Gene therapy—the goal of which is to cure inheritable and acquired diseases by supplying genetic information to various tissues—is a promising therapy of the new millennium. To date, many strategies have been attempted to cure a disease by adding a foreign gene or correcting a mutation in the genes. The success of these gene-therapy strategies is largely dependent on the development of a vector that delivers and efficiently expresses a therapeutic gene in a specific cell population. Viral vectors are potentially efficient, although nonviral vectors have some advantages in that they are typically less immunogenic and easier to prepare. Direct, nonviral gene transfer into the whole organism remains a desirable goal for gene therapy because it avoids laborious and costly cell culture; the gene could be administered as easily as a drug.