Table 2

Using the zebrafish model for gene/protein or small-molecule discovery relevant to human blood disorders

Zebrafish mutant (protein or small molecule)

Human disorder or potential therapeutic application

Known function

Discovery

weissherbst*

(ferroportin)

Hemochromatosis

Iron export^†‡

[21]

frascati* (frs)

(mitoferrin)

Hypochromic anemia

Iron assimilation^†‡

[22]

shiraz* (sir)

(glutaredoxin 5)

Hypochromic anemia

Assembly of Fe-S cluster^†‡

[23]

Dimethyl prostaglandin E2

Bone marrow transplant therapy (clinical trial)

Enhanced HSC engraftment^†

[45]

3F8

GSK3 implicated in type II diabetes, bipolar disorder, Alzheimer's disease, some cancers

Inhibits GSK3^†

[47]

Thiazole-carboxamide 10A

PLK1 upregulated in many cancers

Inhibits PLK1^†

[81]

GSK3, glycogen synthase kinase 3; HSC, hematopoietic stem cell; PLK1, polo-like kinase 1. *Three hematopoietic mutations originally identified in zebrafish have since been linked to human genetic mutation and disease (see text for details). ^†Known function in zebrafish. ^‡Known function in humans.

Martin et al. Genome Medicine 2011 3:83   doi:10.1186/gm299