Human cellular model systems of β-thalassemia enable in-depth analysis of disease phenotype

Abstract β-thalassemia is a prevalent genetic disorder causing severe anemia due to defective erythropoiesis, with few treatment options. Studying the underlying molecular defects is impeded by paucity of suitable patient material. In this study we create human disease cellular model systems for β-t...

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Bibliographic Details
Main Authors: Deborah E. Daniels, Ivan Ferrer-Vicens, Joseph Hawksworth, Tatyana N. Andrienko, Elizabeth M. Finnie, Natalie S. Bretherton, Daniel C. J. Ferguson, A. Sofia. F. Oliveira, Jenn-Yeu A. Szeto, Marieangela C. Wilson, John N. Brewin, Jan Frayne
Format: Article
Language:English
Published: Nature Portfolio 2023-10-01
Series:Nature Communications
Online Access:https://doi.org/10.1038/s41467-023-41961-9