Genome editing by targeted chromosomal mutagenesis.
1Department of Biochemistry, School of Medicine, University of Utah, Emma Eccles Jones Medical Research Building, Rm 4520, 15N. Medical Drive East, Salt Lake City, UT, 84112-5650, USA, dana@biochem.utah.edu.
Methods in Molecular Biology (Clifton, N.J.)
|November 20, 2014
Summary
Genome engineering tools like CRISPR/Cas, TALENs, and ZFNs enable precise DNA modifications in diverse organisms. These advancements hold significant promise for agriculture and human gene therapy applications.
Area of Science:
- Molecular Biology
- Genetics
- Biotechnology
Background:
- Genome engineering tools have advanced significantly, becoming more accessible.
- CRISPR/Cas nucleases, TALENs, and ZFNs allow for targeted DNA double-strand breaks.
Purpose of the Study:
- To review the applications and potential of current genome engineering technologies.
- To highlight the successful use of targeted nucleases across various organisms.
Main Methods:
- Utilizing targeted nucleases (CRISPR/Cas, TALENs, ZFNs) to induce double-strand breaks in DNA.
- Leveraging cellular DNA repair mechanisms for mutation induction and sequence replacement.
Main Results:
- Targeted nucleases have been successfully applied to a wide range of animals, plants, and cultured cells.
- The efficiency and outcomes of genome engineering are system-dependent, influenced by delivery methods and biological context.
Conclusions:
- Genome engineering tools offer powerful capabilities for introducing desirable traits in economically significant organisms.
- The prospects for advancing human gene therapy using these technologies are highly promising.
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