Crispr-mediated human genome editing with vectors
AssigneeRegents of the University of Minnesota
InventorsLi Ou, Chester B. Whitley
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Abstract
Compositions and methods for Cas-based ex vivo and in vivo gene therapy applications are provided.
Claims27
A method to prevent, inhibit or treat a disease in a human, comprising:
- administering to the human an effective amount of i) Cas or an isolated nucleic encoding Cas, and ii) isolated nucleic acid for one or more gRNAs comprising a targeting sequence for a human genomic target and nucleic acid comprising a coding sequence for a prophylactic or therapeutic gene product flanked by homology arms that bind to the human genomic target, or
- an effective amount of iii) isolated nucleic encoding Cas and nucleic acid for one or more gRNAs comprising a targeting sequence for a human genomic target, and iv) isolated nucleic acid comprising a coding sequence for a prophylactic or therapeutic gene product flanked by homology arms that bind to the human genomic target,
- wherein the expression of the coding sequence in the human prevents, inhibits or treats the disease.
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A composition comprising a first vector comprising an isolated nucleic encoding Cas9 and a second vector comprising an isolated nucleic comprising sequences for one or more gRNAs comprising a selected human genomic targeting sequence and a selected coding sequence flanked by homology arms that bind to the human genomic target, or a first vector comprising an isolated nucleic encoding Cas9 and an isolated nucleic comprising sequences for one or more gRNAs comprising a selected human targeting sequence and a second vector comprising a selected coding sequence flanked by homology arms that bind to the human genomic target, wherein at least one of the homolog arms is mutated.
27-34. (canceled)
A method to decrease Cas9 activity on a nucleic acid template having two homology arms specific for a locus in a mammalian genome, comprising:
- introducing into a mammalian cell a nucleic acid template having two homology arms each flanking a nucleotide sequence of interest, wherein at least one of the homology arms is mutated, and wherein the cell comprises Cas9 and a gRNA.
36-46. (canceled)
Dependencies are inferred from the claim text.