The DOI matches its Crossref record; 6 of the 6 references it cites were found in a registry or an index.
megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering
Open accessAbstract
Rare-cleaving endonucleases have emerged as important tools for making targeted genome modifications. While multiple platforms are now available to generate reagents for research applications, each existing platform has significant limitations in one or more of three key properties necessary for therapeutic application: efficiency of cleavage at the desired target site, specificity of cleavage (i.e. rate of cleavage at ‘off-target’ sites), and efficient/facile means for delivery to desired target cells. Here, we describe the development of a single-chain rare-cleaving nuclease architecture, which we designate ‘megaTAL’, in which the DNA binding region of a transcription activator-like (TAL) effector is used to ‘address’ a site-specific meganuclease adjacent to a single desired genomic target site. This architecture allows the generation of extremely active and hyper-specific compact nucleases that are compatible with all current viral and nonviral cell delivery methods.
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Cites
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<tt>BLAT</tt> —The <tt>BLAST</tt> -Like Alignment Tool
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High-Frequency off-Target Mutagenesis Induced by CRISPR-Cas Nucleases in Human Cells
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The mechanism of double-strand DNA break repair by the nonhomologous DNA end-joining pathway
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Therapeutic genome editing: prospects and challenges
Cox, D. B. T., Platt, R. J., & Zhang, F. (2015) · Nature Medicine · doi:10.1038/nm.3793 · Cited by 1,336
Genome-editing Technologies for Gene and Cell Therapy
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Genome-Editing Technologies: Principles and Applications
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Self-citation
Metadata
- Authors
- Boissel, S., Jarjour, J., Astrakhan, A., Adey, A., Gouble, A., Duchateau, P., Shendure, J., Stoddard, B. L., Certo, M. T., Baker, D., & Scharenberg, A. M.
- Venue
- Nucleic Acids Research
- Publisher
- Oxford University Press (OUP)
- Published
- 2013-11-26
- Volume
- 42
- Issue
- 4
- Pages
- 2591-2601
- ISSN
- 0305-1048 (ISSN-L)
- 1362-4962
- OpenAlex
- W2119377684
- Cited by
- 173
- References
- 6
- FWCI
- 9.68
- Type
- journal article
- Language
- en
- Open access
- gold
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- Boissel, S., Jarjour, J., Astrakhan, A., Adey, A., Gouble, A., Duchateau, P., Shendure, J., Stoddard, B. L., Certo, M. T., Baker, D., & Scharenberg, A. M. (2014). megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering. Nucleic Acids Research. https://doi.org/10.1093/nar/gkt1224
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