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 1Ā° transplant | 55% of CD34+ HSPCs | 44% CD34+ HSPCs |
Prior to 1Ā° transplant (16 weeks) | 28% of CD34+ HSPCs | 26% of CD34+ HSPCs |
2Ā° Transplant (32 weeks) | 20% of CD34+ HSPCs | 9.5% of CD34+ HSPCs |