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US 12,667,628 B2Granted
Compositions and methods for gene replacement
AssigneeChildren's Medical Center Corporation
InventorJeffrey R. Holt
Life of this patent
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Abstract
The present invention features a dual vector system for disrupting and replacing a target gene comprising a mutation (e.g., dominant, recessive mutation). Embodiments of the invention may also provide compositions comprising the dual vector system, and methods of using the dual vector system, including but not limited to methods of modifying the genome of a cell, methods of genomic editing, and methods of treating cells or a subject suffering from a genetic disease comprising a mutation.
Claims18
A dual vector system comprising:
- a first vector comprising:
- a polynucleotide encoding a Cas9-KKH polypeptide,
- wherein the first vector does not comprise a polynucleotide encoding a guide RNA (gRNA);
- and
- a second vector comprising:
- a polynucleotide encoding a guide RNA (gRNA) that binds a target gene comprising a mutation, and
- a polynucleotide encoding a wild-type version of the target gene,
- wherein the gRNA is selected from the group consisting of:
- gRNA 12 (SEQ ID NO:
- 15), gRNA 15 (SEQ ID NO:
- 18), and gRNA 16 (SEQ ID NO:
- 23).
9 more elements
A dual vector system comprising:
- a) a first AAV9-PHP.B vector comprising a nucleotide sequence encoding Cas9-KKH,
- wherein the first AAV9-PHP.B vector does not comprise a polynucleotide encoding a guide RNA (gRNA);
- and
- b) a second AAV9-PHP.B vector comprising a nucleotide sequence encoding a guide RNA that binds a TMC1 gene comprising a DFNA36 mutation and a polynucleotide encoding a wild-type TMC1 gene,
- wherein the gRNA is selected from the group consisting gRNA 12 (SEQ ID NO:
- 15), gRNA 15 (SEQ ID NO:
- 18), and gRNA 16 (SEQ ID NO:
- 23).
5 more elements
Dependencies are inferred from the claim text.