Table 1 Deep sequencing analysis of targeted gene editing versus off-target effects in bone marrow cells 15 weeks after a single in utero γtcPNA/DNA nanoparticle (NP) treatment
From: In utero nanoparticle delivery for site-specific genome editing
Gene locus | Sequences of partial homology (5′ to 3′) | Size of region sequenced | Amplicons sequenced | Number modified | Frequency (%) |
|---|---|---|---|---|---|
β-globin | TGCCCTGAAAGAAAGAGA | 128 | 7230901 | 448040 | 6.2 |
Vascular cell adhesion protein precursor 1 | AGCCCTGAAAGAAAGAGA | 111 | 6280254 | 0 | 0 |
Polypyrimidine tract binding protein | GAACCTGAAAGAAAGAGA | 101 | 4452674 | 0 | 0 |
Protocadherin fat 4 precursor | CACCCTGAAAGAAAGAAG | 115 | 5742183 | 0 | 0 |
Olfactory receptor 266 | AAGCCTGAAAGAAAGATT | 172 | 6148312 | 0 | 0 |
Syntaxin-binding protein | AGAAATGAAAGAAAGAGA | 150 | 6660839 | 0 | 0 |
Muscleblind-like protein | GGTGGTGAAAGAAAGAGA | 165 | 5553180 | 0 | 0 |
Ceruloplasmin isoform | AGGACTGAAAGAAAGAGT | 154 | 6197021 | 0 | 0 |
Total off-target | 41034463 | 0 | <0.000002 |
