Table 4 Summary of IL2RGsgRNA off-target INDEL frequency analysis
From: Gene correction for SCID-X1 in long-term hematopoietic stem cells
Gene name | Guide Seq | COSMID | Chromosome location | Features | Expression in HSCsa | U2OS (plasmid) | WT CD34+ 20 nt RNP (WT Cas9) | SCID-X1 CD34+ 19 nt RNP (WT Cas9) | SCID-X1 CD34+ 20 nt RNP (HiFi Cas9) | SCID-X1 CD34+19 nt RNP (HiFi Cas9) |
|---|---|---|---|---|---|---|---|---|---|---|
IL2RG | X | Exon | Yes | 81.1% | 81.7% | 91.7% | 94.1% | 97.6% | ||
LIN01287 | ā | 7 | Intergenic | Data not available | Background | Background | Background | Not sequenced | Not sequenced | |
MPZL1 | ā | 1 | Intron | Yes | 1.1% | 0.1% | 0.1% | Background | Background | |
SHQ1 | ā | ā | 3 | Intron | Yes | 1.5% | Background | Background | Not sequenced | Not sequenced |
SMYD3 | ā | 1 | Intron | Yes | 4.2% | Background | Background | Not sequenced | Not Sequenced | |
ZNF330 | ā | 4 | Intergenic | Data not available | Background | 0.23% | 0.27% | Background | Background |
