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CRISPR-Cas systems and methods for altering expression of gene products, structural information and inducible modular Cas enzymes

机译:CRISPR-CAS系统和改变基因产物表达,结构信息和诱导性模块化CAS酶的表达的方法

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 eukaryotic cells and methods for utilizing the CRISPR-Cas system. In particular the present invention comprehends the engineering of optimized modular CRISPR-Cas enzyme systems.
机译:本发明提供了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,在编码CRISPR复合物的克切者复合体,载体和载体系统的修饰组件中,使用该信息的使用该信息,以及方法 用于设计和使用此类向量和组件。 还提供了在真核细胞中引导Cr Crpr复合体的方法和利用CRISPR-CAS系统的方法。 特别地,本发明理解优化模块化CASP-CAS酶系统的工程。

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