DELIVERY, ENGINEERING AND OPTIMIZATION OF TANDEM GUIDE SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION
    4.
    发明申请
    DELIVERY, ENGINEERING AND OPTIMIZATION OF TANDEM GUIDE SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION 审中-公开
    串联指导系统的交付,工程和优化,序列控制的方法和组合

    公开(公告)号:WO2014204724A8

    公开(公告)日:2015-12-23

    申请号:PCT/US2014041800

    申请日:2014-06-10

    IPC分类号: C12N15/63 C12N9/22 C12N15/10

    摘要: The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences. Provided are vectors and vector systems, some of which encode one or more components of a CRISPR complex, as well as methods for the design and use of such vectors. Also provided are methods of directing CRISPR complex formation in prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity.

    摘要翻译: 本发明提供了用于操纵靶序列的序列和/或活性的系统,方法和组合物的递送,工程和优化。 提供了载体和载体系统,其中一些编码CRISPR复合物的一个或多个组分,以及用于设计和使用这种载体的方法。 还提供了在原核和真核细胞中引导CRISPR复合物形成的方法,以确保增强靶标识别和避免毒性的特异性。

    FUNCTIONAL SCREENING WITH OPTIMIZED FUNCTIONAL CRISPR-CAS SYSTEMS
    5.
    发明申请
    FUNCTIONAL SCREENING WITH OPTIMIZED FUNCTIONAL CRISPR-CAS SYSTEMS 审中-公开
    功能筛选与优化功能CRISPR-CAS系统

    公开(公告)号:WO2016049258A3

    公开(公告)日:2016-06-02

    申请号:PCT/US2015051830

    申请日:2015-09-24

    摘要: The invention provides for systems, methods, and compositions for altering expression of target gene sequences and related gene products. Provided are structural information on the Cas protein of the CRISPR-Cas system, use of this information in generating modified components of the CRISPR complex, vectors and vector systems which encode one or more components or modified components of a CRISPR complex, as well as methods for the design and use of such vectors and components. Also provided are methods of directing CRISPR complex formation in eukaryotie cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends optimized functional CRISPR-Cas enzyme systems.

    摘要翻译: 本发明提供用于改变靶基因序列和相关基因产物的表达的系统,方法和组合物。 提供了关于CRISPR-Cas系统的Cas蛋白的结构信息,使用该信息来生成CRISPR复合物的修饰组分,编码CRISPR复合物的一种或多种组分或修饰组分的载体和载体系统,以及方法 用于设计和使用这些矢量和组件。 还提供了在真核细胞中引导CRISPR复合物形成的方法以及利用CRISPR-Cas系统的方法。 特别地,本发明包括优化的功能性CRISPR-Cas酶系统。

    NUCLEOTIDE-SPECIFIC RECOGNITION SEQUENCES FOR DESIGNER TAL EFFECTORS
    7.
    发明申请
    NUCLEOTIDE-SPECIFIC RECOGNITION SEQUENCES FOR DESIGNER TAL EFFECTORS 审中-公开
    设计师TAL效应的核素特异性识别序列

    公开(公告)号:WO2013082519A3

    公开(公告)日:2014-09-04

    申请号:PCT/US2012067428

    申请日:2012-11-30

    IPC分类号: C12N15/87 C07K14/195

    摘要: The invention relates to methods of altering expression of a genomic locus of interest or specifically targeting a genomic locus of interest in an animal cell, which may involve contacting the genomic locus with a non-naturally occurring or engineered composition that includes a deoxyribonucleic acid (DNA) binding polypeptide having a N-terminal capping region, a DNA binding domain comprising at least five or more Transcription activator-like effector (TALE) monomers and at least one or more half-monomers specifically ordered to target the genomic locus of interest, and a C-terminal capping region, wherein the polypeptide includes at least one or more effector domains, and wherein the polypeptide is encoded by and translated from a codon optimized nucleic acid molecule so that the polypeptide preferentially binds to the DNA of the genomic locus.

    摘要翻译: 本发明涉及改变感兴趣的基因组座位的表达或特异性靶向动物细胞中的基因座的基因座的方法,其可能涉及使基因组座位与包含脱氧核糖核酸(DNA)的非天然存在或工程化组合物接触 )结合多肽,DNA结合结构域包含至少五个或更多个转录激活子样效应子(TALE)单体和至少一种或多种特异性命令靶向感兴趣的基因组座位的半单体,以及 C端封端区,其中所述多肽包括至少一个或多个效应域,并且其中所述多肽由密码子优化的核酸分子编码和翻译,使得所述多肽优先结合所述基因组座位的DNA。