DELIVERY, ENGINEERING AND OPTIMIZATION OF SYSTEMS, METHODS AND COMPOSITIONS FOR SEQUENCE MANIPULATION AND THERAPEUTIC APPLICATIONS
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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.
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Citations
61 Claims
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1-31. -31. (canceled)
- 32. A vector containing nucleic acid(s) encoding inducible Cas9 and at least one nuclear localization signal (NLS), whereby when the vector is delivered to a eukaryotic cell, when induced, Cas9 is expressed in vivo in the eukaryotic cell.
- 53. A Cas9 knock in non-human eukaryote.
Specification