SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION WITH OPTIMIZED FUNCTIONAL CRISPR-CAS SYSTEMS农业专利-农业专业知识服务系统

专利权人:: The Broad Institute, Inc.;PRESIDENT AND FELLOWS OF HARVARD COLLEGE;Massachusetts Institute of Technology;University of Tokyo

发明人:: Silvana KONERMANN,Alexandro TREVINO,Mark BRIGHAM,Fei RAN,Patrick HSU,Chie-yu LIN,Osamu NUREKI,Hiroshi NISHIMASU,Ryuichiro ISHITANI,Feng ZHANG

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 optimized functional CRISPR-Cas enzyme systems.