Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
β-thalassemia is characterised by the presence of an excess of α-globin chains, which contribute to erythrocyte pathology. Here the authors use CRISP/Cas9 to reduce α-globin expression in hematopoietic precursors, and show effectiveness in xenograft assays in mice.
Main Authors: | , , , , , , , , , , , , , , , , , , , , , |
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Format: | Article |
Language: | English |
Published: |
Nature Publishing Group
2017-09-01
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Series: | Nature Communications |
Online Access: | https://doi.org/10.1038/s41467-017-00479-7 |