Title |
Applications of TALENs and CRISPR/Cas9 in Human Cells and Their Potentials for Gene Therapy
|
---|---|
Published in |
Molecular Biotechnology, May 2014
|
DOI | 10.1007/s12033-014-9771-z |
Pubmed ID | |
Authors |
Jingwen Niu, Bin Zhang, Hu Chen |
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. |
X Demographics
Geographical breakdown
Country | Count | As % |
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United States | 1 | 25% |
Unknown | 3 | 75% |
Demographic breakdown
Type | Count | As % |
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Members of the public | 2 | 50% |
Science communicators (journalists, bloggers, editors) | 2 | 50% |
Mendeley readers
Geographical breakdown
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Germany | 2 | 1% |
United States | 2 | 1% |
United Kingdom | 1 | <1% |
Unknown | 151 | 97% |
Demographic breakdown
Readers by professional status | Count | As % |
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Student > Master | 34 | 22% |
Student > Bachelor | 28 | 18% |
Researcher | 27 | 17% |
Student > Ph. D. Student | 23 | 15% |
Other | 6 | 4% |
Other | 18 | 12% |
Unknown | 20 | 13% |
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Biochemistry, Genetics and Molecular Biology | 36 | 23% |
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Neuroscience | 3 | 2% |
Engineering | 3 | 2% |
Other | 10 | 6% |
Unknown | 22 | 14% |