Engineering and optimization of systems, methods, enzymes and guide scaffolds of CAS9 orthologs and variants for sequence manipulation

US11578312B2 · kind B2 · utility

Assignees

• The Broad Institute, Inc. · US
• Massachusetts Institute of Technology · US
• President and Fellows of Harvard College · US
• THE UNIVERSITY OF TOKYO · JP

Inventors

• Feng Zhang · Cambridge, US
• Winston X. Yan · Brookline, US
• Osamu Nureki · Bunkyo, JP
• Kaijie Zheng · Cambridge, US
• Le Cong · Cambridge, US
• Hiroshi Nishimasu · Tokyo, JP
• Fei Ran · Boston, US
• Yinqing Li · Cambridge, US

Key dates

• Filing date: Dec 12, 2017
• Grant date: Feb 14, 2023
• Expiry date: Jul 9, 2041

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2800/80
• WIPO field: Biotechnology
• WIPO sector: Chemistry

Abstract

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. In particular the present invention comprehends engineered new guide architectures and enzymes to be used in optimized Staphylococcus aureus CRISPR-Cas enzyme systems.