Molecular Biotechnology

, Volume 55, Issue 1, pp 54–62 | Cite as

Biological and Biomedical Applications of Engineered Nucleases

  • Yunzhi Pan
  • Li Xiao
  • Alice S. S. Li
  • Xu Zhang
  • Pierre Sirois
  • Jia ZhangEmail author
  • Kai LiEmail author


The development of engineered nucleases is the fruit of a new technological approach developed in the last two decades which has led to significant benefits on genome engineering, particularly on gene therapy. These applications enable efficient and specific genetic modifications via the induction of a double-strand break (DSB) in a specific genomic target sequence, followed by the homology-directed repair (HDR) or non-homologous end joining (NHEJ) pathways. In addition to the application on gene modification in cells and intact organisms, a number of recent papers have reported that this gene editing technology can be applied effectively to human diseases. With the promising data obtained using engineered endonucleases in gene therapy, it appears reasonable to expect that more diseases could be treated and even be cured in this new era of individualized medicine. This paper first brief introduces the development of engineered nucleases with a special emphasis on zinc-finger nucleases (ZFNs) and transcription activator-like effector (TALE) nucleases (TALENs), and then takes CCR5-based gene therapy as an example to discuss the therapeutic applications of engineered nucleases.


Engineered nucleases ZFNs TALENs Gene therapy CCR5 HIV 



This study is partially supported by National Natural Science Foundation of China (No. 30970877), Chinese National 863 Major Grant (No. 2012AA020905), the Priority Academic Program Development of Jiangsu Higher Education Institutions and Innovation Project of Jiangsu Graduate Education (No. CXZZ11_0117).


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Copyright information

© Springer Science+Business Media New York 2012

Authors and Affiliations

  1. 1.Department of Molecular Diagnostics and Biopharmaceutics, College of Pharmaceutical ScienceSoochow UniversitySuzhouChina
  2. 2.University of WaterlooOntarioCanada
  3. 3.Genotheramics Corp.San DiegoUSA
  4. 4.CHUL Research CenterLaval UniversityQuebecCanada

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