Here, we present a systematic approach for developing physiologically relevant, sensitive and specific in vivo assays for interpreting variation in human pathology. Transient genetic manipulation via microinjection of WT and mutant human mRNA and morpholino (MO) antisense oligonucleotides harness the tractability of the developing zebrafish embryo to rapidly assay pathogenic mutations, especially, but not exclusively, in the context of human developmental disorders.
Niederriter, A. R., Davis, E. E., Golzio, C., Oh, E. C., Tsai, I., Katsanis, N. In Vivo Modeling of the Morbid Human Genome using Danio rerio. J. Vis. Exp. (78), e50338, doi:10.3791/50338 (2013).