Table 3. cDNA with exon 53 skipping and INDEL rates in cDNA with exon 53.
% of cDNA with exon 53 skipping | cDNA without exon 53 skipping | ||
---|---|---|---|
Total INDELs (%) | Restoring INDELs (%)a | ||
Muscle tissue | 21.7±6.4 (n = 3) | 0.37±0.12 (n = 3) | ND |
Non-targeting RNP | 54.3 (n = 1) | 1.4 (n = 1) | 1.4 (n = 1)b |
Sa-gRNA2 RNPs | 58.5±3.6 (n = 3) | 5.3±0.95 (n = 3) | 3.5±0.45 (n = 3) |
Sp-gRNA1 RNPs | 56.4± 2.9 (n = 2) | 11.2±1.5 (n = 3) | 6.1±1.0 (n = 3) |
Sp-gRNA2 RNPs | 45.3±4.8 (n = 2) | 25.9±2.2 (n = 3)** | 16.1±2.2 (n = 3)** |
aCalculated based on the top 20 most frequently observed readings, accounting for 85%~95% of all readings.
bThe two types of INDELs accounted for all observed INDELs, all restored dystrophin reading frame.
** indicates p<0.01 when compared with values of Sp-gRNA1 or Sa-gRNA1 (Tukey’s Multiple Comparison Test following ANOVA).