Hooked! Modeling Human Disease in Zebrafish

Overview

Journal J Clin Invest

Specialty General Medicine

Date 2012 Jul 4

PMID 22751109

Citations 225

Authors

Cristina Santoriello

Leonard I Zon

Affiliations

Soon will be listed here.

Abstract

Zebrafish have been widely used as a model system for studying developmental processes, but in the last decade, they have also emerged as a valuable system for modeling human disease. The development and function of zebrafish organs are strikingly similar to those of humans, and the ease of creating mutant or transgenic fish has facilitated the generation of disease models. Here, we highlight the use of zebrafish for defining disease pathways and for discovering new therapies.

Citing Articles

Characterization of a novel zebrafish model of -associated Charcot-Marie-Tooth disease type 4B3.

Lindzon J, List M, Geissah S, Ariaz A, Zhao M, Dowling J Brain Commun. 2025; 7(2):fcaf077.

PMID: 40066109 PMC: 11891516. DOI: 10.1093/braincomms/fcaf077.

Neuroprotective capacity of Celastrus paniculatus on rotenone-induced parkinsonism in zebrafish model.

Manoharan N, Jayamurali D, Sridhar A, Govindarajulu S Mol Biol Rep. 2025; 52(1):272.

PMID: 40024951 DOI: 10.1007/s11033-025-10384-8.

Loss of protein C vs protein S results in discrepant thrombotic phenotypes.

Ku C, Yu X, Zhao Q, Zhao Q, Grzegorski S, Daniel J Blood Adv. 2024; 9(3):545-557.

PMID: 39657127 PMC: 11821410. DOI: 10.1182/bloodadvances.2024013237.

Aligning with the 3Rs: alternative models for research into muscle development and inherited myopathies.

Mehmood H, Kasher P, Barrett-Jolley R, Walmsley G BMC Vet Res. 2024; 20(1):477.

PMID: 39425123 PMC: 11488271. DOI: 10.1186/s12917-024-04309-z.

Exploration of Compounds with 2-Phenylbenzo[]oxazole Scaffold as Potential Skin-Lightening Agents through Inhibition of Melanin Biosynthesis and Tyrosinase Activity.

Jin Jung H, Park H, Soo Park H, Kim H, Yoon D, Park Y Molecules. 2024; 29(17).

PMID: 39275009 PMC: 11396935. DOI: 10.3390/molecules29174162.

References

Sun Z, Amsterdam A, Pazour G, Cole D, Miller M, Hopkins N . A genetic screen in zebrafish identifies cilia genes as a principal cause of cystic kidney. Development. 2004; 131(16):4085-93. DOI: 10.1242/dev.01240. View

Tamary H, Alter B . Current diagnosis of inherited bone marrow failure syndromes. Pediatr Hematol Oncol. 2007; 24(2):87-99. DOI: 10.1080/08880010601123240. View

Wienholds E, Plasterk R . Target-selected gene inactivation in zebrafish. Methods Cell Biol. 2004; 77:69-90. DOI: 10.1016/s0091-679x(04)77004-1. View

Stemple D . TILLING--a high-throughput harvest for functional genomics. Nat Rev Genet. 2004; 5(2):145-50. DOI: 10.1038/nrg1273. View

Li T, Huang S, Zhao X, Wright D, Carpenter S, Spalding M . Modularly assembled designer TAL effector nucleases for targeted gene knockout and gene replacement in eukaryotes. Nucleic Acids Res. 2011; 39(14):6315-25. PMC: 3152341. DOI: 10.1093/nar/gkr188. View

McGrail M, Hatler J, Kuang X, Liao H, Nannapaneni K, Noack Watt K . Somatic mutagenesis with a Sleeping Beauty transposon system leads to solid tumor formation in zebrafish. PLoS One. 2011; 6(4):e18826. PMC: 3080878. DOI: 10.1371/journal.pone.0018826. View

Danilova N, Sakamoto K, Lin S . Ribosomal protein S19 deficiency in zebrafish leads to developmental abnormalities and defective erythropoiesis through activation of p53 protein family. Blood. 2008; 112(13):5228-37. DOI: 10.1182/blood-2008-01-132290. View

Norton N, Li D, Rieder M, Siegfried J, Rampersaud E, Zuchner S . Genome-wide studies of copy number variation and exome sequencing identify rare variants in BAG3 as a cause of dilated cardiomyopathy. Am J Hum Genet. 2011; 88(3):273-82. PMC: 3059419. DOI: 10.1016/j.ajhg.2011.01.016. View

Lieber M . The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway. Annu Rev Biochem. 2010; 79:181-211. PMC: 3079308. DOI: 10.1146/annurev.biochem.052308.093131. View

10.

Shafizadeh E, Paw B, Foott H, Liao E, Barut B, Cope J . Characterization of zebrafish merlot/chablis as non-mammalian vertebrate models for severe congenital anemia due to protein 4.1 deficiency. Development. 2002; 129(18):4359-70. DOI: 10.1242/dev.129.18.4359. View

11.

Patton E, Widlund H, Kutok J, Kopani K, Amatruda J, Murphey R . BRAF mutations are sufficient to promote nevi formation and cooperate with p53 in the genesis of melanoma. Curr Biol. 2005; 15(3):249-54. DOI: 10.1016/j.cub.2005.01.031. View

12.

Poss K, Wilson L, Keating M . Heart regeneration in zebrafish. Science. 2002; 298(5601):2188-90. DOI: 10.1126/science.1077857. View

13.

Tobin J, Beales P . Restoration of renal function in zebrafish models of ciliopathies. Pediatr Nephrol. 2008; 23(11):2095-9. PMC: 7462901. DOI: 10.1007/s00467-008-0898-7. View

14.

Donovan A, Brownlie A, Zhou Y, Shepard J, Pratt S, Moynihan J . Positional cloning of zebrafish ferroportin1 identifies a conserved vertebrate iron exporter. Nature. 2000; 403(6771):776-81. DOI: 10.1038/35001596. View

15.

Bassett D, Currie P . The zebrafish as a model for muscular dystrophy and congenital myopathy. Hum Mol Genet. 2003; 12 Spec No 2:R265-70. DOI: 10.1093/hmg/ddg279. View

16.

Taylor A, Humphries J, White R, Murphey R, Burns C, Zon L . Hematopoietic defects in rps29 mutant zebrafish depend upon p53 activation. Exp Hematol. 2011; 40(3):228-237.e5. PMC: 3310385. DOI: 10.1016/j.exphem.2011.11.007. View

17.

Gieger C, Radhakrishnan A, Cvejic A, Tang W, Porcu E, Pistis G . New gene functions in megakaryopoiesis and platelet formation. Nature. 2011; 480(7376):201-8. PMC: 3335296. DOI: 10.1038/nature10659. View

18.

Avery-Kiejda K, Bowden N, Croft A, Scurr L, Kairupan C, Ashton K . P53 in human melanoma fails to regulate target genes associated with apoptosis and the cell cycle and may contribute to proliferation. BMC Cancer. 2011; 11:203. PMC: 3120805. DOI: 10.1186/1471-2407-11-203. View

19.

Wienholds E, van Eeden F, Kosters M, Mudde J, Plasterk R, Cuppen E . Efficient target-selected mutagenesis in zebrafish. Genome Res. 2003; 13(12):2700-7. PMC: 403812. DOI: 10.1101/gr.1725103. View

20.

Urnov F, Rebar E, Holmes M, Zhang H, Gregory P . Genome editing with engineered zinc finger nucleases. Nat Rev Genet. 2010; 11(9):636-46. DOI: 10.1038/nrg2842. View