THERAPEUTIC PROMISE OF MOLECULAR-GENETICS

The application of molecular genetics to medicine is based on the obse rvation that even common diseases have both genetic and environmental components. Conventional medicines are often effective in managing env ironmental components of disease but are generally ineffective in mana ging genetic diseases or manipulating the genetic component of multifa ctorial diseases. The development of therapies aimed at the genetic co mponent of disease will require non-conventional medicinal application s of molecular genetics. Various approaches have been proposed such as diagnosing the propensity for disease to facilitate early interventio n with conventional therapies, selectively eliminating mutant genes fr om human populations, correcting mutations in human chromosomes, and u sing genes as medicines to modify the genetic components of disease. O f these, it is the development of gene medicines that has the greatest practical potential. The combination of conventional medicines, focus ed on the environmental components of disease, and gene medicines, foc used on the genetic components, will provide the clinician with broad options for managing health and disease. The challenge to molecular bi ology is to develop gene medicines that are effective, safe, and socia lly acceptable, and therapies that map well to established clinical pr actice and may be employed efficaciously alongside conventional medici nes. .