【摘 要】
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Ribosomopathy is a group of human diseases that are caused by aberrant ribosome functions.Patients of ribosomopathy normally present themselves with bone ma
【机 构】
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DepartmentofMolecular,Cell,andDevelopmentalBiology,UniversityofCaliforniaatLosAngeles,LosAngeles,Cal
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Ribosomopathy is a group of human diseases that are caused by aberrant ribosome functions.Patients of ribosomopathy normally present themselves with bone marrow failure, which ultimately leads to severe anemia.Other common features in patients include a shorter stature and a higher predisposition to certain cancers.We model two ribosome-associated disorders in the zebrafish, DBA and 5q-sydrome, via targeted mutations of RPS19, RPL11 and RPS14, respectively.Using these models, we show that a delay in late-stage erythropoiesis occurs and is followed by cell death upon ribosome stress.The delay in late-stage erythropoiesis is independent of p53 signaling and may, in part, be due to overexpression of Lft1.We demonstrated the ability of RAP-011, an activin receptor type ⅡA ligand trap, to increase erythropoiesis in the zebrafish and restore erythroid levels in our zebrafish models.Furthermore, the effects of RAP-011 are independent of p53 signaling, and thus, offer a new strategy in the treatment of ribosomopathies.
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