Zebrafish PKD-targeted Services

The zebrafish is the premier nonmammalian vertebrate model organism that has a kidney, and it is well-established as a model system for the study of PKD and other ciliopathies.

For PKD research, the zebrafish model can be used to understand the molecular and cellular basis of the disease, assess the functional role of human genetic variations, and screen for small-molecule suppressors in PKD zebrafish embryonic disease models.

Services provided by the Model Systems Core are available to PKD researchers both at Mayo and other institutions. Services are supported and complemented by Mayo's Zebrafish Core Facility, which provides animal housing and extensive technical expertise.


Five zebrafish PKD-targeted services are offered by the Model Systems Core:

  • Custom TALEN synthesis for targeted gene mutations. Assistance is provided with TALEN design and synthesis. Training is available on mutation screening and line development.
  • Small-molecule screening for cystic phenotype effects. A recent screen for small-molecule suppressors of a PKD zebrafish embryonic disease model has demonstrated the viability of using zebrafish to explore chemical genetics questions. Also, one major emphasis in PKD is the potential use of combinatorial treatment approaches, so the zebrafish offers an opportunity to explore new small molecules as well as combinations of known drugs for preclinical efficacy testing.
  • Evaluation of pathways relevant to PKD. For example, microinjection of mRNAs or antisense morpholino oligonucleotides into embryos. The zebrafish embryo enables rapid in vivo testing of specific hypotheses related to PKD disease formation and progression.
  • Human sequence variation functional testing. The zebrafish can be used to discern functional differences in human sequence variation of candidate PKD loci. The Zebrafish Core Facility provides necessary expertise to run comparable in vivo functional tests for human sequence variants identified in the Translational PKD Center.
  • Targeted training on current zebrafish technologies. The core offers training on zebrafish-related topics of interest to Translational PKD Center faculty and scientists.

Available lines

Line Description Reference
Cup tc321 Pkd2 mutant Schottenfeld, et al., 2007; Brand, et al., 1996; Haffter, et al., 1996
Enpep-GFP Pronephric marker Seiler and Pack, 2010
hi4166 Pkd2 mutant Sun, et al., 2004
Locke Cystic mutant Brand, et al., 1996; Haffter, et al., 1996
Na pump-GFP Pronephric marker Liu, et al., 2007
Switch hitter Cilia mutant Brand, et al., 1996; Haffter, et al., 1996; Sullivan-Brown, 2007
WT1b-GFP Pronephric marker Perner, et al., 2007
GBT0046 Pronephric marker Petzold, et al., 2009; Clark, et al., 2011

Lines may be available for distribution upon request.


For more information about zebrafish services offered through the Model Systems Core, contact the core.