Massachusetts Institute of Technology

The Broad Institute, Inc.

Harvard College

The University of Tokyo

University of Iowa Research Foundation

Chie-Yu Lin

Feng Zhang

Patrick D Hsu

Fei Ran

Beverly L Davidson

Silvana Konermann

Osamu Nureki

Hiroshi Nishimasu

Edgardo Rodriguez

Mark Daniel Brigham

Ryuichiro Ishitani

Alexandro Trevino

The invention provides for delivery, engineering and optimization of systems, methods, and compositions for manipulation of sequences and/or activities of target sequences especially for use as to nucleotide repeat disorders. Provided are delivery systems and tissues or organ which are targeted as sites for delivery especially for use as to nucleotide repeat disorders. Also provided are vectors and vector systems some of which encode one or more components of a CRISPR complex or system especially for use as to nucleotide repeat disorders, as well as methods for the design and of such. Also provided are methods of directing CRISPR complex or system formation in eukaryotic cells especially for use as to nucleotide repeat disorders including with consideration of specificity for target recognition and avoidance of toxicity and editing or modifying a target site in a genomic locus of interest to alter or improve the status of a disease or a condition.

Compositions and methods of use of CRISPR-Cas systems in nucleotide repeat disorders

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.

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

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 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 prokaryotic and eukaryotic cells to ensure enhanced specificity for target recognition and avoidance of toxicity.

Optimized CRISPR-Cas double nickase systems, methods and compositions for sequence manipulation

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Cambridge, MA, United States of America

Chie-Yu Lin