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AssigneesThe Broad Institute, Inc., Massachusetts Institute of Technology, President and Fellows of Havard College
InventorsFeng Zhang, Le Cong, Fei Ran, Matthias Heidenreich, Lukasz Swiech
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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. 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.
An AAV1, AAV2, AAV3, AAV4, AAV5, AAV6, AAV8, AAV9, AAV1/2, or AAV2/8 viral vector that contains heterologous nucleic acid molecule(s) engineered for expression in a eukaryotic cell, wherein the heterologous nucleic acid molecule(s) encodes a Cas9 and a CRISPR-Cas system guide, wherein the Cas9 and the system guide are capable of forming a CRISPR-Cas complex after expression in the eukaryotic cell, wherein the system guide is capable of targeting the complex to a DNA sequence in the genome of the eukaryotic cell,
Dependencies are inferred from the claim text.