Healing the Liver: Cell and Gene Therapies for Inherited and Acquired Diseases

The liver plays a fundamental role in maintaining homeostasis thanks to the numerous functions performed by this organ. Non-inherited metabolic liver diseases, inherited metabolic liver diseases, and liver cancers are pathological conditions affecting liver function and that can lead to its failure. To date, for end-stage liver diseases—where the remaining hepatic tissue is no longer capable of regenerating sufficiently rapidly—or for metabolic diseases involving the liver, liver transplantation remains the standard and ideal therapeutic approach. However, this is limited by donor availability, surgical costs, and the tangible risk of autoimmune rejection, which may occur at varying intervals post-surgery. Furthermore, for the duration of their lives, transplant recipients must undergo systemic immunosuppressive treatment to prevent rejection; this is associated with high costs and severe side effects, including infections and secondary malignancies. In this review, we discuss these pathologies and how recent cell-based therapies and/or gene therapy approaches have emerged as promising alternatives that can provide either temporary restoration of hepatic function or long-term benefits, potentially reducing the global burden of liver disorders.