Gene Therapy for Liver Disease

Liver plays a critical role in many inherited and acquired genetic disorders. It is also the prime location for the treatment of certain inborn errors of metabolism. Over the years, advances in nucleic acid-based therapies have resulted in several successful treatment outcomes for liver disorders. However, their use has been severely hampered by many undesirable side effects and methodological restraints. To address these issues, recent research efforts have been focused on the development of targeted gene editing approaches using novel genetic tools, as well as the efficient delivery systems for these tools into the liver. This chapter will provide an overview on current and emerging therapeutic strategies for liver-directed gene therapy.