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Mol Biotechnol. 2014 Aug;56(8):681-8. doi: 10.1007/s12033-014-9771-z.

Applications of TALENs and CRISPR/Cas9 in human cells and their potentials for gene therapy.

Author information

1
Department of Hematopoietic Stem Cell Transplantation, Affiliated Hospital of Academy of Military Medical Sciences, No. 8 Dongda Street, Fengtai District, Beijing, 100071, People's Republic of China.

Abstract

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.

PMID:
24870618
DOI:
10.1007/s12033-014-9771-z
[Indexed for MEDLINE]

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