Table 1.
Subject | Gene modification | Efficiency in neonates (embryos) | Off-target mutation | Mosaicism | Genome editing | Remarks | Reference |
---|---|---|---|---|---|---|---|
Mouse zygotes | Introduction of V5 tag (42bp) into Sox2, two loxP (34 bp) sites into Mecp2 | Sox: 6.0%, Mecp2: 0.8% | Yes (Mecp2) | Yes | Cas9 | Cytoplasmic or pronuclear injection | [39] |
Mouse zygotes | Correction of Crytg with 1bp deletion in exon3 | 4.4∼5.7% | Yes | N.D. | Cas9 | Cytoplasmic injection | [40] |
Mouse zygotes | Correction of Dmdmdx | 9.1% | (No)a | Yes | Cas9 | Pronuclear injection only, or pronuclear and cytoplasmic injections | [41] |
Mouse zygotes | Correction of Crb1rd8 | 27% | Yes | Yes | TALEN | Pronuclear injection | [42] |
Rat zygotes | Correction of Tyrc, Asipa, Kit h | Tyr: 7.7 %, Asip:18.2%, Kit: 4.0% | (No)a | Yes | Cas9 | Pronuclear injection | [43] |
Human zygotes | Introduction of silent mutations into HBB | (4.7%) | Yes | Yes | Cas9 | Cytoplasmic injection | [24] |
(No) shows that no off-target mutations were identified at potential off-target sites.