Therapeutic promise of molecular genetics

The application of molecular genetics to medicine is based on the observation that even common diseases have both genetic and environmental components. Conventional medicines are often effective in managing environmental components of disease but are generally ineffective in managing genetic diseases or manipulating the genetic component of multifactorial diseases. The development of therapies aimed at the genetic component of disease will require non-conventional medicinal applications of molecular genetics. Various approaches have been proposed such as diagnosing the propensity for disease to facilitate early intervention with conventional therapies, selectively eliminating mutant genes from human populations, correcting mutations in human chromosomes, and using genes as medicines to modify the genetic components of disease. Of these, it is the development of gene medicines that has the greatest practical potential. The combination of conventional medicines, focused on the environmental components of disease, and gene medicines, focused on the genetic components, will provide the clinician with broad options for managing health and disease. The challenge to molecular biology is to develop gene medicines that are effective, safe, and socially acceptable, and therapies that map well to established clinical practice and may be employed efficaciously alongside conventional medicines.