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Eltrombopag Improves Erythroid Differentiation in a Human Induced Pluripotent Stem Cell Model of Diamond Blackfan Anemia.

Cells - 26 Mar 2021

Qanash Husam, Li Yongqin, Smith Richard H, Linask Kaari, Young-Baird Sara, Hakami Waleed, Keyvanfar Keyvan, Choy John S, Zou Jizhong, Larochelle Andre

Abstract excerpt

Diamond Blackfan Anemia (DBA) is a congenital macrocytic anemia associated with ribosomal protein haploinsufficiency. Ribosomal dysfunction delays globin synthesis, resulting in excess toxic free heme in erythroid progenitors, early differentiation arrest, and pure red cell aplasia. In this study, DBA induced pluripotent stem cell (iPSC) lines were generated from blood mononuclear cells of DBA patients with...

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Identifiers

PMID: 33810313
DOI: 10.3390/cells10040734
PMCID: PMC8065708

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