Table 2. Single guide RNA profiles in myoblastsa.
DMD 50 sgRNAs | DMD 53 Sa-gRNA2 | DMD 53 Sp-gRNA1 | DMD 53 Sp-gRNA2 | |||||
---|---|---|---|---|---|---|---|---|
LVLP-RNPs (ng p24) | 400 | 400 | 100 | 200 | 200 | 100 | 200 | 400 |
Total Readings | 80718 | 95391 | 39032 | 44434 | 41688 | 36037 | 41562 | 88076 |
Readings with INDELs | 95 | 109 | 512 | 998 | 1137 | 870 | 4987 | 10294 |
P (Chi square) | N/A | 0.89 | <0.0001 | <0.0001 | <0.0001 | <0.0001 | <0.0001 | <0.0001 |
Total INDEL % | 0.12% | 0.11% | 1.3% | 2.24% | 2.7% | 2.4% | 4.8% | 11.7% |
Restoring (Relative %)b | N/A | N/A | 28.5% | 37.6% | 26.7% | 53.8% | 52.0% | 49.9% |
aIndicated amounts of lentiviral capsid packaged Cas9 ribonucleoprotein (ng p24) were delivered to 1x105 myoblasts isolated from hDMDdel52/mdx mice. The target sequences of human DMD exon 53 amplified from gDNA were analyzed by NGS.
bSignificantly different between Sp-gRNA1 and Sp-gRNA2 (t-test, p<0.05).