Gene correction for SCID-X1 in long-term hematopoietic stem cells.
Pavel-Dinu M, Wiebking V, Dejene BT, Srifa W, Mantri S, Nicolas CE, Lee C, Bao G, Kildebeck EJ, Punjya N, Sindhu C, Inlay MA, Saxena N, DeRavin SS, Malech H, Roncarolo MG, Weinberg KI, Porteus MH
Nat Commun. 2019 Apr 9;10(1):1634. doi: 10.1038/s41467-019-09614-y.
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