Translational pig models for human diseases.

Abstract

The translation of novel discoveries in biomedical research into clinical applications is a lengthy and resource-intensive process, with variable success. Predictive model organisms for non-clinical research are a key asset in this process. Rodents remain the most widely used models, but they have limitations in recapitulating human pathophysiology and disease phenotypes. Over the past two decades, pigs have played an increasingly important role in bridging this translational gap. Given their close anatomical and physiological similarity to humans, most vividly illustrated by successful pig-to-human organ xenotransplantation, pigs provide unparalleled advantages for translational research. Advances in genetic engineering now allow the precise recreation of human disease mechanisms in pigs, generating tailored large-animal models with exceptional clinical relevance. This review highlights the potential of genetically modified pig models for human diseases, with a focus on metabolic disorders and rare monogenic diseases such as Duchenne muscular dystrophy, cystic fibrosis, and Laron syndrome.