Molecular Biotechnology

, Volume 56, Issue 8, pp 681–688

Applications of TALENs and CRISPR/Cas9 in Human Cells and Their Potentials for Gene Therapy


DOI: 10.1007/s12033-014-9771-z

Cite this article as:
Niu, J., Zhang, B. & Chen, H. Mol Biotechnol (2014) 56: 681. doi:10.1007/s12033-014-9771-z


The newly developed TALENs and emerging CRISPR/Cas9 have spurred interests in the field of genome engineering because of their ease of customization and high-efficient site-specific cleavages. Although these novel technologies have been successfully used in many types of cells, it is of great importance to apply them in human-derived cells to further observe and evaluate their clinical potentials in gene therapy. Here, we review the working mechanism of TALEN and CRISPR/Cas9, their effectiveness and specificity in human cells, and current methods to enhance efficiency and reduce off-target effects. Besides, CCR5 gene was chosen as a target example to illustrate their clinical potentials. Finally, some questions are raised for future research and for researchers to consider when making a proper choice bases on different purposes.


TALENs CRISPR/Cas9 Genome editing Gene therapy CCR5 

Copyright information

© Springer Science+Business Media New York 2014

Authors and Affiliations

  1. 1.Department of Hematopoietic Stem Cell TransplantationAffiliated Hospital of Academy of Military Medical SciencesBeijingPeople’s Republic of China
  2. 2.Cell and Gene Therapy CenterAcademy of Military Medical SciencesBeijingPeople’s Republic of China

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