Zebrafish DBA models. Wild-type embryos show a high density of red blood cells (WT), whereas the blood production is drastically reduced in the rps19 knockdown (KD) and knockout (KO) embryos, as indicated by the absence of hemoglobin stained cells (lower panels). These embryos also display developmental deformities in head and tail regions (arrowheads). The abnormal phenotypes were rescued by injection of rps19 mRNA but not rescued by mRNA with patient type mutations in the knockdown embryos [14].

Zebrafish in vivo phenotype screening. Zebrafish provides a novel platform for screening compound libraries and evaluating drug efficacy in vivo, which will lead to find new therapeutics for rare diseases including DBA.