Genetic modification and screening in rat using haploid embryonic stem cells

Cell Stem Cell. 2014 Mar 6;14(3):404-14. doi: 10.1016/j.stem.2013.11.016. Epub 2013 Dec 19.

Abstract

The rat is an important animal model in biomedical research, but practical limitations to genetic manipulation have restricted the application of genetic analysis. Here we report the derivation of rat androgenetic haploid embryonic stem cells (RahESCs) as a tool to facilitate such studies. Our approach is based on removal of the maternal pronucleus from zygotes to generate androgenetic embryos followed by derivation of ESCs. The resulting RahESCs have 21 chromosomes, express pluripotency markers, differentiate into three germ layer cells, and contribute to the germline. Homozygous mutations can be introduced by both large-scale gene trapping and precise gene targeting via homologous recombination or the CRISPR-Cas system. RahESCs can also produce fertile rats after intracytoplasmic injection into oocytes and are therefore able to transmit genetic modifications to offspring. Overall, RahESCs represent a practical tool for functional genetic studies and production of transgenic lines in rat.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Base Sequence
  • Biomarkers / metabolism
  • CRISPR-Cas Systems / genetics
  • Cell Differentiation / genetics
  • DNA Transposable Elements / genetics
  • Embryo, Mammalian / metabolism
  • Embryonic Stem Cells / cytology
  • Embryonic Stem Cells / metabolism*
  • Gene Expression Regulation
  • Gene Targeting
  • Genetic Testing*
  • Genome
  • Haploidy*
  • Homologous Recombination / genetics
  • Homozygote
  • Male
  • Mice
  • Molecular Sequence Data
  • Mutation / genetics
  • Pluripotent Stem Cells / cytology
  • Pluripotent Stem Cells / metabolism
  • Rats
  • Sperm Injections, Intracytoplasmic

Substances

  • Biomarkers
  • DNA Transposable Elements