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Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia

Abstract:

β-Thalassemia is one of the most common inherited anemias, with no effective cure for most patients. The pathophysiology reflects an imbalance between α- and β-globin chains with an excess of free α-globin chains causing ineffective erythropoiesis and hemolysis. When α- thalassemia is co-inherited with β-thalassemia, excess free α-globin chains are reduced significantly ameliorating the clinical severity. Here we show the use of CRISPR/Cas9 genome editing of primary human hematopoietic stem/p...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's version

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Publisher copy:
10.1038/s41467-017-00479-7

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Institution:
University of Oxford
Department:
Oxford, MSD, RDM, Weatherall Insti. of Molecular Medicine
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, RDM, Weatherall Insti. of Molecular Medicine
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, RDM, Weatherall Insti. of Molecular Medicine
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, RDM, Weatherall Insti. of Molecular Medicine
More by this author
Institution:
University of Oxford
Department:
Oxford, MSD, RDM, Weatherall Insti. of Molecular Medicine
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Grant:
MC_UU_12025/MC_UU_12009/4
Publisher:
Springer Nature Publisher's website
Journal:
Nature Communications Journal website
Volume:
8
Pages:
424
Publication date:
2017-09-04
Acceptance date:
2017-06-07
DOI:
EISSN:
2041-1723
ISSN:
2041-1723
Pubs id:
pubs:698620
URN:
uri:894b01d4-5485-40bf-8d46-8748c110671c
UUID:
uuid:894b01d4-5485-40bf-8d46-8748c110671c
Local pid:
pubs:698620
Keywords:

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