Systems, methods and compositions for sequence manipulation with optimized functional CRISPR-Cas systems

US12258595B2 · kind B2 · utility

Assignees

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

Inventors

• Silvana Konermann · Menlo Park, US
• Alexandro Trevino · Stanford, US
• Mark Brigham · Cambridge, US
• Fei Ran · Boston, US
• Patrick Hsu · Cambridge, US
• Chie-yu Lin · Boston, US
• Osamu Nureki · Bunkyo, JP
• Hiroshi Nishimasu · Tokyo, JP
• Ryuichiro ISHITANI · Tokyo, JP
• Feng Zhang · Cambridge, US

Key dates

• Filing date: Dec 27, 2022
• Grant date: Mar 25, 2025
• Expiry date: Oct 19, 2043

Classification

• Technology area (CPC Y): Emerging Cross-Sectional Technologies
• CPC primary: Y02A50/30
• WIPO field: Pharmaceuticals
• 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.