The vector copy number in peripheral blood ranged from 0.10 to 1.55 at month 24 (assessed in 14 patients) and from 0.36 to 1.83 at month 36 (assessed in 3 patients) (Figure 1A); the number had ...
Allogeneic hematopoietic stem-cell transplantation for X-linked severe combined immunodeficiency (SCID-X1) often fails to reconstitute immunity associated with T cells, B cells, and natural killer (NK ...