Characterization and Phenotypic Assessment of Zebrafish Disease Models for Splice Modulation Studies

Zebrafish has emerged as a powerful model in biomedical research as it recapitulates many human disease pathologies. Zebrafish share approximately 70% evolutionarily conserved genes with humans. Recent advances in genome editing have enabled many precision and inducible models that mimic human disorders to be rapidly generated for phenotypic analysis. The large clutch size, ex vivo development, and ability to uptake drug compounds through their skin and gills during early larvae development make the zebrafish an extremely attractive system for high-throughput drug screening. Here, we describe the generation of zebrafish models for splice modulation studies and general phenotypic assessments in human disease modeling.