Molecular medicine: a primer for clinicians--part IX human gene therapy

Previous papers in our Molecular Medicine series have described how many tools of the molecular biologist are being used to develop practical bedside applications of modern molecular biology. We have discussed the development of molecular diagnostics and their emerging use in clinical settings. In this paper we discuss how the tools of the molecular biologist are being used to develop "gene therapy", genetic treatments or cures for a wide variety of medical conditions. Recent results suggest that effective gene-based treatments or cures for many human diseases may soon be practical.