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