Editing an alpha-globin enhancer in primary human hematopoietic stem cells as a treatment for beta-thalassemia.
Mettananda S, Fisher CA, Hay D, Badat M, Quek L, Clark K, Hublitz P, Downes D, Kerry J, Gosden M, Telenius J, Sloane-Stanley JA, Faustino P, Coelho A, Doondeea J, Usukhbayar B, Sopp P, Sharpe JA, Hughes JR, Vyas P, Gibbons RJ, Higgs DR
Nat Commun. 2017 Sep 4;8(1):424. doi: 10.1038/s41467-017-00479-7.
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