Table 1 On-target and off-target analyses by next generation sequencing (NGS).
From: Hemophilia A ameliorated in mice by CRISPR-based in vivo genome editing of human Factor VIII
On-Target/ Off-Target | gRNA motif (mismatches in bold font) | PAM (NNGRRT) | Chromosome Position | Strand | Gene | Site position | Indel % | |
|---|---|---|---|---|---|---|---|---|
Vehicle (n = 3) | AAVs (n = 5) | |||||||
On-target | GTTAGAAATACTTGCAAAGCC | TAGAAT | Chr5:90474937–90474963 | + | Alb | intronic | 0 | 9.38 ± 0.90 |
Off-target | GTTTGAAATACTTTTAAAGCC | GTGAGT | Chr5:3757359-3757385 | − | Ankib1 | intronic | 0 | 0 |
Off-target | TTTAGAAATACTTAAAAAGCC | AAGAGT | Chr8:77780734-77780759 | + | NA | intergenic | 0 | 0.58 ± 0.58 |
Off-target | GATAGAAACACTTGCAAA-CC | ATGAAT | Chr8:127199445-127199470 | − | Pard3 | intronic | 0 | 1.84 ± 0.76 |
Off-target | GTTAAGA-CACTTGCAAAGCC | AAGGAT | Chr11:54929261-54929286 | + | Tnip1 | intronic | 0 | 0 |
Off-target | GTTAGAAATACCTGCAAAGC- | TAAGGT | Chr12:7607521-7607546 | + | NA | intergenic | 0 | 0 |
Off-target | GTTAAAAACACTTGCAGAAGCC | CTGGGT | Chr12:111780123-111780150 | − | Klc1 | intronic | 0 | 0 |
Off-target | TTTAGAAATACTAGC-AAGCC | TGGGAT | Chr19:28248970-28248995 | − | NA | intergenic | 0 | 0 |
Off-target | GTTAGAAATTCTTGGAATGCC | TAGGGT | ChrX:15412386-15412412 | + | NA | intergenic | 0 | 0 |
