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

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 wi...

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Bibliographic Details
Published in:	Nature communications Vol. 8; no. 1; pp. 424 - 11
Main Authors:	Mettananda, Sachith, Fisher, Chris A., Hay, Deborah, Badat, Mohsin, Quek, Lynn, Clark, Kevin, Hublitz, Philip, Downes, Damien, Kerry, Jon, Gosden, Matthew, Telenius, Jelena, Sloane-Stanley, Jackie A., Faustino, Paula, Coelho, Andreia, Doondeea, Jessica, Usukhbayar, Batchimeg, Sopp, Paul, Sharpe, Jacqueline A., Hughes, Jim R., Vyas, Paresh, Gibbons, Richard J., Higgs, Douglas R.
Format:	Journal Article
Language:	English
Published:	London Nature Publishing Group UK 04.09.2017 Nature Publishing Group Nature Portfolio
Subjects:	631/337/4041/3196 631/532/1542 692/699/1541/13 alpha-Globins - genetics Animals Antigens, CD34 - metabolism Base Sequence beta-Thalassemia - genetics beta-Thalassemia - therapy Cells, Cultured CRISPR-Cas Systems Enhancer Elements, Genetic - genetics Female Gene Editing Gene Knockdown Techniques Genome editing Genome, Human Hematopoietic Stem Cells - metabolism Heterografts Humanities and Social Sciences Humans Mice multidisciplinary Science Science (multidisciplinary) Sequence Deletion - genetics Single-Cell Analysis Stem cell transplantation Stem cells Thalassemia Xenografts
ISSN:	2041-1723, 2041-1723
Online Access:	Get full text
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