Cell therapy in congenital inherited hepatic disorders

Abstract

Congenital inherited hepatic disorders (CIHDs) are a set of diverse and heterogeneous group of genetic disorders leading to a defect in an enzyme or transporter. Most of these disorders are currently treated by liver transplantation as standard of care. Improved surgical techniques and post-operative care has led to a wider availability and success of liver transplantation program worldwide. However liver transplantation has its own limitations due to invasive surgery and lifelong use of immunosuppressive agents. Our experience from auxiliary liver transplantation (where right or the left lobe of the patient liver is replaced with a healthy liver donor) demonstrated successful treatment of the underlying defect of noncirrhotic metabolic disorder suggesting that whole liver replacement may not be necessary to achieve a change in phenotype. Large number of animal studies in human models of CIHD have shown success of hepatocyte transplantation leading to its human use. This review addresses the current state of human hepatocyte transplantation in the management of CIHDs with bottlenecks to its wider application and future perspectives.

Keywords: Cell therapy; Congenital inherited hepatic disorders; Human hepatocyte transplantation; Liver transplantation.