In Vivo Gene Therapy via Receptor-Mediated DNA Delivery

Human gene therapy has advanced during the past ten years from a theoretical concept to a rapidly emerging technology. Tremendous technological strides in recombinant DNA methodologies have fostered molecular studies in such fields as regulation of gene expression, human genetics and disease states, and gene transfer techniques. The confluence of these fields has led to the emergence of gene therapy as a reality, albeit limited at present to a small number of clinical studies (Anderson, 1992; Miller, 1992). As practiced today most gene therapy protocols lie within the realm of high-cost, technology-intensive, individualized treatments, akin to such procedures as organ and bone marrow transplantation (Mulligan, 1991). This type of procedure, though beneficial, is limited in usefulness since it is not readily accessible to much of the population. Even for those to whom this type of gene therapy strategy would be available, the current inability to achieve permanent transgene expression necessitates periodic retreatments, making these procedures highly cost-ineffective over the long term.