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Table 1 Genome editing of mammalian embryonic stem cells

From: International regulatory landscape and integration of corrective genome editing into in vitro fertilization

Subject Targeted Gene Efficiency of Modification* Off-target Mutation Genome Editing Delivery Method Ref.
NHEJ for InDel
Rat haploid ESCs Tet1, Tet2, Tet3 91% (Single locus), 45% (Double), 14% (Triple) N.D. Cas9 Plasmid/sgRNA [53]
Mouse ESCs Tet1, Tet2, Tet3, Sry, Uty 56 ~ 77% (Single; Tet1,2,3), 21% (Triple; Tet1,2,3) No Cas9 Plasmid [43]
HDR for Gene Addition
Human ESCs GFP into CCR5 5.3% Yes ZFNs Lentivirus [49]
Human ESCs PGK-HygroR into, PIG-A 0.19 ~ 83.49% N.D. ZFNs Plasmid [52]
Human ESCs GFP (or PURO) into, AAVS1, OCT4, PITX 2.9 ~ 15.2% (AAVS1), 0% (OCT4, PITX3) No (AAVS1, OCT4), Yes (PITX3) ZFNs Plasmid [50]
Human ESCs GFP (and/or PURO), into. AAVS1, OCT4, PITX3 9 ~ 22% (AAVS1), 1 ~ 2% (PITX3), 0% (OCT4) Yes (AAVS1, OCT4), N.D. (PITX3) TALENs Plasmid [51]
HDR for Gene Correction or Modification
Human ESCs Removal of Integrated GFP 0.24% N.D. ZFNs Plasmid [52]
Human ESCs Introduction of HindIII cleavage site into EMX1 0.4% No Cas9 nickase mutant Plasmid/sgRNA/Oligo [42]
Mouse ESCs Crytg with 1 bp deletion in exon3 0 ~ 44.4% Yes Cas9 Plasmid [35]
  1. ESCs; embryonic stem cells, NHEJ; non-homologous end-joining, HDR; homology-directed repair, GFP; green fluorescent protein, PURO; puromycin, sgRNA; single guide RNA, N.D.; not determined.
  2. *Biallelic modification.