Journal article
Editing an α-globin enhancer in primary human hematopoietic stem cells as a treatment for β-thalassemia
- Abstract:
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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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(pdf, 2.6MB)
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- Publisher copy:
- 10.1038/s41467-017-00479-7
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Authors
Funding
Bibliographic Details
- 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:
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2041-1723
- ISSN:
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2041-1723
- Pubs id:
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pubs:698620
- URN:
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uri:894b01d4-5485-40bf-8d46-8748c110671c
- UUID:
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uuid:894b01d4-5485-40bf-8d46-8748c110671c
- Local pid:
- pubs:698620
Item Description
- Keywords:
Terms of use
- Copyright holder:
- Mettananda et al.
- Copyright date:
- 2017
- Notes:
- Author(s) retain copyright; published by Nature Publishing Group under a Creative Commons Attribution 4.0 International License.
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