Engineering nucleases for gene targeting: safety and regulatory considerations.

N Biotechnol

Scientific Institute of Public Health, Biosafety and Biotechnology Unit, J. Wytsmanstraat 14, Brussels B-1050, Belgium. Electronic address:

Published: January 2014

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.

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http://dx.doi.org/10.1016/j.nbt.2013.07.001DOI Listing

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