Related Experiment Video
Updated: May 9, 2026
12:04
Mouse Genome Engineering Using Designer Nucleases
Published on: April 2, 2014
Engineering nucleases for gene targeting: safety and regulatory considerations
Katia Pauwels1, Nancy Podevin, Didier Breyer
1Scientific Institute of Public Health, Biosafety and Biotechnology Unit, J. Wytsmanstraat 14, Brussels B-1050, Belgium.
New Biotechnology
|July 16, 2013
Summary
Nuclease-based gene targeting (NBGT) offers broad applications in genetic modification across species. Research highlights safety improvements and regulatory considerations for this powerful genome editing technology.
Area of Science:
- Genetics and Molecular Biology
- Biotechnology
- Bioengineering
Background:
- Nuclease-based gene targeting (NBGT) is a versatile genome editing technology with applications spanning from microorganisms to humans.
- The rapid advancement of NBGT and custom nucleases has led to increased data on potential off-target effects.
- Understanding these effects is crucial for the safe and effective application of NBGT.
Purpose of the Study:
- To discuss the potential of NBGT for genetic modification.
- To identify key aspects for improving the safety of NBGT techniques.
- To review the regulatory oversight concerning NBGT applications and products.
Main Methods:
- Literature review and synthesis of current research on NBGT.
- Analysis of off-target effects associated with NBGT.
- Discussion of safety enhancement strategies for NBGT.
- Examination of regulatory frameworks for NBGT applications.
Main Results:
- NBGT is applicable across a wide range of organisms, including those of economic importance.
- Growing data indicate the presence of off-target effects in NBGT.
- Several strategies can be employed to improve the safety profile of NBGT techniques.
- Regulatory oversight is an important consideration for the implementation of NBGT.
Conclusions:
- NBGT presents a significant advancement in genome editing with broad applicability.
- Addressing off-target effects is critical for enhancing the safety and reliability of NBGT.
- Appropriate regulatory frameworks are necessary to guide the responsible use of NBGT technologies.
Related Concept Videos
01:16In-vitro Mutagenesis
To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
00:49What is Genetic Engineering?
Overview
01:59CRISPR
Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced Short...
02:53Conservative Site-specific Recombination and Phase Variation
Because the DNA segments are cut and reorganized in a direction-specific manner, site-specific recombination has emerged as an efficient genetic engineering technique. Flippase and Cyclization recombinases or Flp and Cre, respectively, are two members of the tyrosine recombinase family derived from bacteriophages, that are used to mediate site-specific DNA insertions, deletions, and targeted expression of proteins in mammalian cell lines.
The recognition sites for Cre recombinase called LoxP...
The recognition sites for Cre recombinase called LoxP...