[Therapeutic drug screening with zebrafish models]

The zebrafish mutant strains to mimic human diseases have been developed to study human diseases and to discover novel therapeutic drugs. The characteristics of zebrafish, small size, large clutch size, ex utero development, body transparency, and genetic tractability are very useful to research approaches like therapeutic drug screening. Here we introduce and discuss zebrafish models of human diseases and methods of drugs screening using them. Specifically, we show examples of therapeutic drug screens using zebrafish models of a muscular dystrophy to identify new candidates to improve phenotypes in skeletal muscle. The drug screening also reveals new drug actions and unexpected directions for future therapeutic ways. Phenotypic drug screen using zebrafish is an important for rapidly developing and validating therapeutics for human diseases.