Future Prospects of Gene Therapy for Treating CNS Diseases

Abstract

In the last several decades, enormous gains have been made in our understanding of the pathogenesis of various human diseases. By understanding the biochemical, molecular, and genetic mechanisms through which a disease state manifests itself, it becomes possible to develop rational strategies for therapeutic intervention. In addition to providing insight into disease mechanisms, studies using molecular biology have provided a tool, gene therapy, through which it is possible to induce a cell to make a specific protein that could interfere with the disease process. Gene therapy provides for a local, regulated delivery of the therapeutic gene product, thereby avoiding some of the systemic side effects of drug therapies. Gene therapy has been investigated with some success in several organ systems and this review discusses efforts to use gene therapy in central nervous system (CNS) diseases. Considerations for using gene therapy and the benefits of various delivery approaches are addressed. The potential efficacy and drawbacks of gene therapy for CNS disease are discussed in light of advances in preclinical research. Finally, the future prospects for the use of gene therapy in the clinic are discussed in terms of safety, ethical considerations, and public opinion.