Generation of a monkey with MECP2 mutations by TALEN-based gene targeting

Neurosci Bull. 2014 Jun;30(3):381-6. doi: 10.1007/s12264-014-1434-8. Epub 2014 May 16.

Abstract

Gene editing in model organisms has provided critical insights into brain development and diseases. Here, we report the generation of a cynomolgus monkey (Macaca fascicularis) carrying MECP2 mutations using transcription activator-like effector nucleases (TALENs)-mediated gene targeting. After injecting TALENs mRNA into monkey zygotes achieved by in vitro fertilization and embryo transplantation into surrogate monkeys, we obtained one male newborn monkey with an MECP2 deletion caused by frameshifting mutation in various tissues. The monkey carrying the MECP2 mutation failed to survive after birth, due to either the toxicity of TALENs or the critical requirement of MECP2 for neural development. The level of MeCP2 protein was essentially depleted in the monkey's brain. This study demonstrates the feasibility of introducing genetic mutations in non-human primates by site-specific gene-editing methods.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Endonucleases / genetics
  • Female
  • Fertilization in Vitro
  • Gene Targeting* / methods
  • HEK293 Cells
  • Humans
  • Macaca fascicularis
  • Male
  • Methyl-CpG-Binding Protein 2 / genetics*
  • Methyl-CpG-Binding Protein 2 / metabolism
  • Microinjections
  • Mutation / genetics*
  • Oocytes
  • RNA, Messenger / metabolism
  • Transcription Factors / genetics
  • Transcription Factors / metabolism*
  • Transfection
  • Zygote / physiology

Substances

  • MECP2 protein, human
  • Methyl-CpG-Binding Protein 2
  • RNA, Messenger
  • Transcription Factors
  • Endonucleases