Using Zebrafish to Study Human Genetic Disease

Abstract Zebrafish is becoming a more and more popular model organism to study human genetic disease. Recent advances in genome editing, including but not limited to the CRISPR/cas technology, have made zebrafish model one of the fastest, cheapest and easiest model to generate mutations similar to the ones identified in human genetic disorders. Based on its size, zebrafish mutant embryos are also a perfect model to be used in chemical preclinical screens to identify novel molecules that are able to compensate for the partial or total inactivation of a gene. Progress in human genome sequencing has recently identified several novel alleles linked to genetic disorders that can now be tested for the identification of novel therapeutics in a zebrafish mutant modelled to phenocopy the human mutation. Key Concepts Zebrafish is a remarkable model to study human genetic diseases The zebrafish genetic tool box has a novel reliable tool: the CRISPR/cas9 technology for reverse genetics Zebrafish as a model for drug discovery Development of zebrafish as a now tool for cure in personalised/precision medicine