Delivery, use and therapeutic applications of the CRISPR-Cas systems and compositions for genome editing

US11407985B2 · kind B2 · utility

Assignees

• The Broad Institute, Inc. · US
• Massachusetts Institute of Technology · US

Inventors

• Le Cong · Cambridge, US
• David Benjamin Turitz Cox · Cambridge, US
• Matthias Heidenreich · Cambridge, US
• Randall Jeffrey Platt · Cambridge, US
• Lukasz Swiech · Boston, US
• Feng Zhang · Cambridge, US

Key dates

• Filing date: Jun 10, 2016
• Grant date: Aug 9, 2022
• Expiry date: Jun 10, 2036

Classification

• Technology area (CPC C): Chemistry; Metallurgy
• CPC primary: C12N2750/14143
• WIPO field: Pharmaceuticals
• WIPO sector: Chemistry

Abstract

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 delivery systems and tissues or organ which are targeted as sites for delivery. Also 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 eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity and to edit or modify a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.