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    • Gene Correction for SCID-X1 In Long-Term Hematopoietic Stem Cells 

      Pavel-Dinu, M.; Wiebking, V.; Dejene, B. T.; Srifa, W.; Mantri, S.; Nicolas, C. E.; Lee, C.; Bao, G.; Kildebeck, Eric J.; Punjya, N.; Sindhu, C.; Inlay, M. A.; Saxena, N.; DeRavin, S. S.; Malech, H.; Roncarolo, M. G.; Weinberg, K. I.; Porteus, M. H.
      Gene correction in human long-term hematopoietic stem cells (LT-HSCs) could be an effective therapy for monogenic diseases of the blood and immune system. Here we describe an approach for X-linked sSevere cCombined ...