Streamlined ex vivo and in vivo genome editing in mouse embryos using recombinant adeno-associated viruses.

ABSTRACT: Recent advances using CRISPR-Cas9 approaches have dramatically enhanced the ease for genetic manipulation in rodents. Notwithstanding, the methods to deliver nucleic acids into pre-implantation embryos have hardly changed since the original description of mouse transgenesis more than 30 years ago. Here we report a novel strategy to generate genetically modified mice by transduction of CRISPR-Cas9 components into pre-implantation mouse embryos via recombinant adeno-associated viruses (rAAVs). Using this approach, we efficiently generated a variety of targeted mutations in explanted embryos, including indel events produced by non-homologous end joining and tailored mutations using homology-directed repair. We also achieved gene modification in vivo by direct delivery of rAAV particles into t

SUBMITTER: Yoon Y 

PROVIDER: S-EPMC5788975 | biostudies-literature | 2018 Jan

REPOSITORIES: biostudies-literature