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

 

    Sachith Mettananda, Chris A. Fisher, Deborah Hay, Mohsin Badat, Lynn Quek, Kevin Clark, Philip Hublitz, Damien Downes, Jon Kerry, Matthew Gosden, Jelena Telenius, Jackie A. Sloane-Stanley, Paula Faustino, Andreia Coelho, Jessica Doondeea, Batchimeg Usukhbayar, Paul Sopp, Jacqueline A. Sharpe, Jim R. Hughes, Paresh Vyas, Richard J. Gibbons & Douglas R. Higgs

 

    Nature Communications volume 8, Article number: 424 (2017)

 

    doi:10.1038/s41467-017-00479-7

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