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...
Main Authors: | , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Portfolio
2023-10-01
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Series: | Nature Communications |
Online Access: | https://doi.org/10.1038/s41467-023-41961-9 |