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AssigneesThe Broad Institute, Inc., Massachusetts Institute of Technology, University of Iowa Research Foundation
InventorsBeverly Davidson, Chie-yu Lin, Edgardo Rodriguez, Feng Zhang
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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.
An engineered composition for in vivo genome editing in a multicellular organism, comprising at least two CRISPR-Cas system guides and at least one Cas9 protein, or one or more vectors encoding the at least two CRISPR-Cas system guides and the at least one Cas9 protein, wherein the at least two CRISPR-Cas system guides are engineered to target at least two CRISPR-Cas complexes to at least two DNA sequences in a eukaryotic cell that together flank a nucleotide sequence comprising a defective nucleotide element, repeat or expansion to thereby mediate cleavage of the flanking DNA sequences and excision of the defective nucleotide element, repeat or expansion, and
An engineered composition for in vivo genome editing in a multicellular organism, comprising an AAV vector encoding at least two CRISPR-Cas system guides and at least one Cas9 protein, wherein the at least two CRISPR-Cas system guides are engineered to target at least two CRISPR-Cas complexes to at least two DNA sequences in a eukaryotic cell that together flank a nucleotide sequence comprising a defective nucleotide element, repeat or expansion to thereby mediate cleavage of the flanking DNA sequences and excision of the defective nucleotide element, repeat or expansion, and
Dependencies are inferred from the claim text.