Engineering Xenopus embryos for phenotypic drug discovery screening

摘要: Many rare human inherited diseases remain untreatable despite the fact that disease causing genes are known and adequate mouse models have been developed. In vivo phenotypic drug screening relies on isolating candidates by their ability to produce a desired therapeutic phenotype in whole organisms. Embryos of zebrafish Xenopus frogs abundant, small free-living. They can be easily arrayed multi-well dishes treated with organic molecules. With development novel genome modification tools, such zinc-finger nucleases (ZFNs), transcription activator-like effector (TALENs), CRISPR/Cas, it is now possible efficiently engineer non-mammalian diseases. Here, we will review rapid progress made adapting these editing tools Xenopus. The advantages embryos as study presented utility for discovery discussed. Being tetrapod, complements an indispensable animal model pathologies therapeutics