In vitro and in vivo translational models for rare liver diseases

Highlights

• Targeting early steps of liver disease progression can lead to more successful therapeutic interventions.

• Validated animal models reflective of human liver disease phenotypes increase predictability of drug toxicity and efficacy.

• 3D liver tissue models are being developed as in vitro platforms for drug development and regenerative medicine.

Abstract

A challenge in developing effective treatments is the modeling of the human disease using in vitro and in vivo systems. Animal models have played a critical role in the understanding of disease pathophysiology, target validation, and evaluation of novel therapeutic agents. However, as the success rate from entry into clinical testing to drug approval remains low, it is critical to have high quality and well-validated models reflective of the disease condition. Additional experimental models are being developed based on functional in vitro 3D tissue models such as organoids and 3D bioprinted tissues. Because these 3D tissue models mimic closer the architecture, cell composition and physiology of native tissues, they are now being used as screening platforms in drug discovery and development and for tissue transplant in regenerative medicine. Here we review the current state-of-art of in vitro and in vivo translational models for the development of therapies for rare diseases of the liver.