megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering.
megaTALs: a rare-cleaving nuclease architecture for therapeutic genome engineering.
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DOI：
10.1093/nar/gkt1224
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发表时间：
2014-02
影响因子：
14.9
通讯作者：
Scharenberg AM
中科院分区：
文献类型：
--
作者：
Boissel S;Jarjour J;Astrakhan A;Adey A;Gouble A;Duchateau P;Shendure J;Stoddard BL;Certo MT;Baker D;Scharenberg AM
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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影响因子：
14.9
作者：
Li T;Huang S;Jiang WZ;Wright D;Spalding MH;Weeks DP;Yang B
通讯作者：
Yang B
影响因子：
56.9
作者：
Jinek, Martin;Chylinski, Krzysztof;Charpentier, Emmanuelle
通讯作者：
Charpentier, Emmanuelle
影响因子：
56.9
作者：
Boch, Jens;Scholze, Heidi;Bonas, Ulla
通讯作者：
Bonas, Ulla
DOI：
10.1126/science.1232033
发表时间：
2013-02-15
期刊：
Science (New York, N.Y.)
影响因子：
--
作者：
Mali P;Yang L;Esvelt KM;Aach J;Guell M;DiCarlo JE;Norville JE;Church GM
通讯作者：
Church GM
影响因子：
12.4
作者：
Cornu, Tatjana I.;Thibodeau-Beganny, Stacey;Cathomen, Toni
通讯作者：
Cathomen, Toni