title: Highly efficient RNA-guided base editing in mouse embryos.
abstract: Base editors (BEs) composed of a cytidine deaminase fused to CRISPR-Cas9 convert cytidine to uridine, leading to single-base-pair substitutions in eukaryotic cells. We delivered BE mRNA or ribonucleoproteins targeting the Dmd or Tyr gene via electroporation or microinjection into mouse zygotes. F0 mice showed nonsense mutations with an efficiency of 44-57% and allelic frequencies of up to 100%, demonstrating an efficient method to generate mice with targeted point mutations.
authors.affiliation: Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.
authors.forename: Kyoungmi
authors.initials: K
authors.lastname: Kim
authors.affiliation: Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.|Department of Chemistry, Seoul National University, Seoul, Republic of Korea.
authors.forename: Seuk-Min
authors.initials: SM
authors.lastname: Ryu
authors.affiliation: Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.
authors.forename: Sang-Tae
authors.initials: ST
authors.lastname: Kim
authors.affiliation: Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.
authors.forename: Gayoung
authors.initials: G
authors.lastname: Baek
authors.affiliation: Department of Chemistry, Seoul National University, Seoul, Republic of Korea.
authors.forename: Daesik
authors.initials: D
authors.lastname: Kim
authors.affiliation: Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.|Department of Chemistry, Seoul National University, Seoul, Republic of Korea.
authors.forename: Kayeong
authors.initials: K
authors.lastname: Lim
authors.affiliation: Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.|Department of Chemistry, Seoul National University, Seoul, Republic of Korea.
authors.forename: Eugene
authors.initials: E
authors.lastname: Chung
authors.affiliation: Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.|Department of Chemistry, Seoul National University, Seoul, Republic of Korea.
authors.forename: Sunghyun
authors.initials: S
authors.lastname: Kim
authors.affiliation: Center for Genome Engineering, Institute for Basic Science, Seoul, Republic of Korea.|Department of Chemistry, Seoul National University, Seoul, Republic of Korea.
authors.forename: Jin-Soo
authors.initials: JS
authors.lastname: Kim
mesh_terms: D000818:Animals; D020029:Base Pairing / Q000235:genetics; D064130:CRISPR-Associated Proteins / Q000235:genetics*; D064112:Clustered Regularly Interspaced Short Palindromic Repeats / Q000235:genetics*; D003564:Cytidine Deaminase / Q000235:genetics; D004622:Embryo, Mammalian / Q000196:embryology / Q000502:physiology*; D000072669:Gene Editing / Q000379:methods*; D051379:Mice; D008810:Mice, Inbred C57BL; D008813:Mice, Inbred ICR; D016297:Mutagenesis, Site-Directed / Q000379:methods*; D017354:Point Mutation / Q000235:genetics; D012313:RNA / Q000235:genetics*; D011993:Recombinant Fusion Proteins / Q000235:genetics
chemical_list: D064130:CRISPR-Associated Proteins; D011993:Recombinant Fusion Proteins; D012313:RNA; D003564:Cytidine Deaminase