Efficient targeting of FATS at a common fragile site in mice through TALEN-mediated double-hit genome modification

Biotechnol Lett. 2014 Mar;36(3):471-9. doi: 10.1007/s10529-013-1387-z. Epub 2013 Oct 25.

Abstract

Transcription activator-like effector nucleases (TALENs) have emerged as a newly developed approach for genome editing. However, its application in targeting specific genomic loci susceptible to DNA damage remains obscure. Here, we report a modified approach for TALENs-based targeting of FATS, a fragile-site gene whose major introns have AT-rich sequence and di-nucleotide repeats. Two pairs of FATS-TALENs were designed to cleave two sites specifically at a coding exon of FATS. After in vitro transcription, the mRNA from FATS-TALEN pairs was microinjected into mouse zygotes. The targeting efficiency of two FATS-TALEN pairs in vivo was more than threefold higher than that of one FATS-TALEN pair. Moreover, large-size DNA deletions were detected, which were heritable and easily detectable by PCR. Our study indicates that the double-hit TALEN approach enhances targeting efficiency in vivo and provides convenience for monitoring germline transmission of mutations by PCR, which will facilitate the functional research on fragile-site genes.

Publication types

  • Research Support, Non-U.S. Gov't

MeSH terms

  • Animals
  • Endonucleases / metabolism*
  • Gene Targeting / methods*
  • Mice
  • Mice, Inbred C57BL
  • Microinjections

Substances

  • Endonucleases