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. 2020 Sep 24;15(9):e0239468. doi: 10.1371/journal.pone.0239468

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).