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. © 2014 Springer Science+Business Media.
CITATION STYLE
Niu, J., Zhang, B., & Chen, H. (2014). Applications of TALENs and CRISPR/Cas9 in human cells and their potentials for gene therapy. Molecular Biotechnology. Humana Press Inc. https://doi.org/10.1007/s12033-014-9771-z
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