New Innovations: Therapies for Genetic Conditions

Abstract

New, mechanistic understanding of genetic disease is prompting the rapid advancement of treatments for these diseases. Treatment strategies for monogenic hereditary disorders and complex genetic disorders are evolving with novel uses of traditional medications, targeted therapies for biochemical deficiencies including enzyme replacement, and pathology-targeted pharmacologic approaches to enhance normal function. There is also tangible progress in the development of nucleic acid-derived therapies, utilizing both DNA and RNA to modify gene expression and to correct a genetic disease process. Technologies that use antisense oligonucleotides, RNA interference, gene transfer, and stem cell transplantation are resulting in promising treatment breakthroughs. Though serious challenges remain in the safe and successful implementation of these technologies in clinical practice, the exciting prospect of treatment for previously untreatable genetic conditions is now within sight, providing hope for both patients and providers. Current options and evolving therapies are reviewed for the broad category of genetic disease.