Table 2 IL2RG cDNA genome targeted frequencies pre- and pos-t transplant

From: Gene correction for SCID-X1 in long-term hematopoietic stem cells

Time of genome editing quantification (ddPCR)

% Functionally corrected cells (BM) IH engraftment

% Functionally corrected cells (BM) IF engraftment

Prior to 1° transplant

55% of CD34+ HSPCs

44% CD34+ HSPCs

Prior to 1° transplant (16 weeks)

28% of CD34+ HSPCs

26% of CD34+ HSPCs

2° Transplant (32 weeks)

20% of CD34+ HSPCs

9.5% of CD34+ HSPCs

  1. Percent homologous direct repair (HDR) as measured by ddPCR at the indicated time points. CD34+ HSPCs are derived from healthy male donors. Median shown. 1° transplant: IH (n = 10), IF (n = 14). 2° transplant: IH (n = 8), IF (n = 12)
Back to article page