Xenopus: An Undervalued Model Organism to Study and Model Human Genetic Disease

Abstract

The function of normal and defective candidate genes for human genetic diseases, which are rapidly being identified in large numbers by human geneticists and the biomedical community at large, will be best studied in relevant and predictive model organisms that allow high-speed verification, analysis of underlying developmental, cellular and molecular mechanisms, and establishment of disease models to test therapeutic options. We describe and discuss the pros and cons of the frog Xenopus, which has been extensively used to uncover developmental mechanisms in the past, but which is being underutilized as a biomedical model. We argue that Xenopus complements the more commonly used mouse and zebrafish as a time- and cost-efficient animal model to study human disease alleles and mechanisms.

Keywords: Analysis of human disease alleles; Cilia; Ciliopathy; Congenital heart disease; Disease modeling; Left-right asymmetry; Model organisms of human disease; Xenopus.