<b><i>Xenopus</i></b>: 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.

Antibodies, Nanobodies, or Aptamers—Which Is Best for Deciphering the Proteomes of Non-Model Species?

“…Proteins known to be expressed in humans were also found to be enriched in frogs, but surprisingly not in mice or pigs [35]. It is unsurprising then that frogs may find their way into more translatable research in the future [36], especially given that half of human genes differ from their mouse orthologs in different developmental trajectories, including more than 200 disease genes associated with brain, heart, and liver diseases [37]. These differences can impact the proteomes, and therefore phenotypes, between humans and mice.…”

Rare Human Diseases: Model Organisms in Deciphering the Molecular Basis of Primary Ciliary Dyskinesia

“…Moreover, research conducted using model organisms can help us to understand the molecular basis of the ciliary defects triggered by PCD-causative mutations. Several unicellular and multicellular organisms have emerged as models to study ciliopathies, for review [40][41][42][43][44].…”

“…Interestingly, due to the presence of both multiciliated cells and mucus-secreting goblet cells, the embryo's skin, to some extent, resembles the epithelium lining the mammalian respiratory tracts [58]. Equally importantly, in the case of both models, the microscopic methods are well developed, and gene manipulation and genome editing are possible [44,[59][60][61][62][63][64][65].…”

A nanobody-based fluorescent reporter reveals human α-synuclein in the cell cytosol

“…As Rpn10 is a well conserved protein across different species (Figure 1b), we presumed that our proposed mechanism should also operate in a model organism that lacks endogenous hαSyn and possesses the conserved Rpn10 epitope recognized by NbSyn87. Accordingly, we chose to validate the proposed mechanism in living Xenopus laevis tadpoles, a time-and cost efficient model organism 72 .…”