Engineering nucleases for gene targeting: safety and regulatory considerations

N Biotechnol. 2014 Jan 25;31(1):18-27. doi: 10.1016/j.nbt.2013.07.001. Epub 2013 Jul 12.


Nuclease-based gene targeting (NBGT) represents a significant breakthrough in targeted genome editing since it is applicable from single-celled protozoa to human, including several species of economic importance. Along with the fast progress in NBGT and the increasing availability of customized nucleases, more data are available about off-target effects associated with the use of this approach. We discuss how NBGT may offer a new perspective for genetic modification, we address some aspects crucial for a safety improvement of the corresponding techniques and we also briefly relate the use of NBGT applications and products to the regulatory oversight.

Publication types

  • Review

MeSH terms

  • Deoxyribonucleases*
  • Gene Targeting*
  • Genetic Therapy* / legislation & jurisprudence
  • Genetic Therapy* / standards
  • Humans
  • Safety* / legislation & jurisprudence
  • Safety* / standards


  • Deoxyribonucleases