Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells

Dual α-globin-truncated erythropoietin receptor knockin restores hemoglobin production in α-thalassemia-derived erythroid cells

Summary: The most severe form of α-thalassemia results from loss of all four copies of α-globin. Postnatally, patients face challenges similar to β-thalassemia, including severe anemia and erythrotoxicity due to the imbalance of β-globin and α-globin chains. Despite progress in genome editing treatm...

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Main Authors: Simon N. Chu, Eric Soupene, Devesh Sharma, Roshani Sinha, Travis McCreary, Britney Hernandez, Huifeng Shen, Beeke Wienert, Chance Bowman, Han Yin, Benjamin J. Lesch, Kun Jia, Kathleen A. Romero, Zachary Kostamo, Yankai Zhang, Tammy Tran, Marco Cordero, Shota Homma, Jessica P. Hampton, James M. Gardner, Bruce R. Conklin, Tippi C. MacKenzie, Vivien A. Sheehan, Matthew H. Porteus, M. Kyle Cromer
Format: Article
Language:English
Published: Elsevier 2025-01-01
Series:Cell Reports
Subjects:
CP: Stem cell research
Online Access:http://www.sciencedirect.com/science/article/pii/S221112472401492X
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