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Table 2 Genome editing of mammalian zygotes

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

Subject Targeted Gene Efficiency in Embryos* Efficiency in Neonates** Off-target Mutation Genome Editing Delivery Method Remarks Ref.
NHEJ for InDel
Monkey, zygotes Nr0b1,Ppar-γ, Rag1 18.2 ~ 40.9%, (Single), 9.1 ~ 27.3% (Double) - No Cas9 mRNA/sgRNA A set of twin female monkeys with modified Rag1 and Ppar-γ were born. [33]
Monkey, zygotes MECP2 - 9.5% (Rhesus), 3.7% (Cynomolgus) No TALENs Plasmid Three miscarried rhesus and cynomologus male fetuses had Mecp2 mutations. [34]
Monkey, zygotes MECP2 - (2.0%) N.D. TALENs mRNA A modified male monkey appeared to be mosaic. [47]
Bovine, zygotes LDLR 3.8% - N.D. TALENs mRNA Cytoplasmic injection [46]
Porcine, zygotes RELA 0.5% - N.D. TALENs mRNA Cytoplasmic injection [46]
Rat, zygotes IgM - 3.9 ~ 5.5% (mRNA) No (Plasmid), Yes (mRNA) TALENs Plasmid or mRNA Mosaic mutations occurred. Plasmid; Pronuclear, mRNA; Cytoplasmic injection. [39]
Rat, Zygotes Tet1,Tet2,Tet3 - 14.3 ~ 18.8% (Double; Tet1,2), 18.6% (Triple) Yes (Triple) Cas9 mRNA/sgRNA Mosaic mutations occurred. Cytoplasmic injection [41]
Mouse, Zygotes Tet1,Tet2,Tet3, Sry, Uty - 8.0 ~ 17.6% (Single), 14.7 ~ 15.3% (Double; Tet1,2) No (Tet1,Tet2), N.D. (Tet3) Cas9 mRNA/sgRNA Pronuclear injection [43]
Mouse, zygotes Mecp2 80 ~ 100% - No Cas9, nickase mutant mRNA/sgRNA Cytoplasmic injection [42]
Mouse, zygotes Exo1 1.4 ~ 6.8% 0 ~ 10.3% N.D. TALENs mRNA Pronuclear injection [48]
Mouse, zygotes Fgf10 - 14.3 ~ 41.7% N.D. Cas9 mRNA/sgRNA Cytoplasmic injection [45]
Mouse, zygotes Fgf10 - 1.3 ~ 1.5% N.D. TALENs mRNA/sgRNA Cytoplasmic injection [45]
HDR for Gene Addition
Mouse, zygotes mCherry into Nanog, GFP into Oct4 - 1.7% (Nanog), 3.0% (Oct4) Yes, (Nanog, Oct4) Cas9 mRNA/sgRNA/Plasmid Cytoplasmic or pronuclear injection [40]
HDR for Gene Correction or Modification
Mouse, zygotes Addition of V5 to Sox2, lox to Mecp2 - 6.0% (Sox2), 0.8% (Mecp2) Yes (Mecp2) Cas9 mRNA/sgRNA/Oligo Cytoplasmic or pronuclear injection [40]
Mouse, zygotes Crytg with 1 bp deletion in exon3 - 4.4 ~ 5.7% Yes Cas9 mRNA/sgRNA/ Oligo Cytoplasmic injection [35]
Mouse, zygotes Introduction of a STOP codon into Fah - 2.0% (wild type), 2.0% (nickase mutant) Yes, (mutant < WT) Cas9 WT and mutant mRNA/sgRNA/Oligo Pronuclear injection [44]
  1. *Genetically modified embryos per injected zygote (%). **Genetically modified neonates (including fetus) per transferred embryo (%). †Genetically modified blastocysts per blastocyst which underwent Cas9 treatment (%).