Peering through zebrafish to understand inherited bone marrow failure syndromes.

Peering through zebrafish to understand inherited bone marrow failure syndromes. Haematologica. 2018 Dec 20;: Authors: Oyarbide U, Topczewski J, Corey SJ Abstract Inherited bone marrow failure syndromes are experiments of nature characterized by impaired hematopoiesis with cancer and leukemia predisposition. The mutations associated with inherited bone marrow failure syndromes affect fundamental cellular pathways, such as DNA repair, telomere maintenance, or proteostasis. How these disturbed pathways fail to produce sufficient blood cells and lead to leukemogenesis are not understood. The rarity of inherited cytopenias, paucity of affected primary human hematopoietic cells, and sometime inadequacy of murine or induced pluripotential stem cell models constitute obstacles to greater knowledge of them. Zebrafish proffer an organismal model to study gene functions. As vertebrates, zebrafish share with humans many orthologous genes involved in blood disorders. As a model organism, zebrafish provides advantages, including rapid development of transparent embryos, high fecundity providing large numbers of mutant and normal siblings, and a large collection of mutant and transgenic lines useful in interrogating the blood system and other tissues during development. Importantly, recent advances in genomic editing in zebrafish can speedily validate the new genes or novel variants discovered in clinical investigation as causes for marrow failure...
Source: Haematologica - Category: Hematology Authors: Tags: Haematologica Source Type: research