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Gene disruption. Primate Model
• Coinjection of Cas9 mRNA and sgRNAs into one-cell- stage cynomolgus monkey embryos.
• Simultaneous disruption of two target genes (Ppar-g and Rag1) in one step, and no off-target mutagenesis was detected by comprehensive analysis.
Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos. Niu, 2014
PCC Genetics - ESHRE BCN 2018
Disease model. Primate Model
• Generation of an immunodeficient nonhuman primate model
• Optimization of ZFN and TALEN targeting of IL2RG gene in marmoset to edit fibroblasts and embryos
• Single blastomere splitting from an 8-cell embryo efficiently identified activated ZFN and TALEN and reduced mosaicism
• IL2RG KO marmosets phenotypes were similar to that of human X- SCID patients
Generation of a nonhuman primate model of severe combined immunodeficiency using highly efficient genome editing. Sato, 2106
PCC Genetics - ESHRE BCN 2018
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