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US 10,648,002 B2Granted
Method for correcting a genetic sequence
AssigneeRegents of the University of Minnesota
InventorsBeau R. Webber, Mark J. Osborn, Jakub Tolar, Bruce R. Blazar
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Abstract
Methods of gene correction, methods of generating induced pluripotent stem cells (iPSCs), and methods of deriving multi-lineage cell types with therapeutic value. In some embodiments, the gene correction affects the expression and/or function of the functional type VII collagen protein (C7).
Claims20
A method comprising:
- introducing into a cell that comprises a genomic sequence in need of editing:
- a donor template polynucleotide that comprises a polynucleotide that encodes an edited version of the genomic sequence;
- a polynucleotide that encodes a clustered regularly interspaced short palindromic repeat associated (Cas) nuclease or nickase;
- and
- a guide RNA (gRNA), wherein the gRNA comprises at least one of:
- (SEQ ID NO:
- 43)
- GTGCTGGGCTTCATAGTTCTTGG,
- (SEQ ID NO:
- 44)
- GGAGGCTGCGTGCTGGGGGCAGG,
- and
- (SEQ ID NO:
- 45)
- GCCTTGGGGTCCAGGGCTTCCGG;
- allowing the nuclease or nickase to cut at least one strand of the genomic sequence;
- and
- allowing the edited version of the genomic sequence to replace the genomic sequence in need of editing to produce a cell comprising a donor sequence.
15 more elements
Dependencies are inferred from the claim text.