Polynucleotides, compositions, and methods for genome editing
AssigneeIntellia Therapeutics, Inc.
InventorsChristian Dombrowski, Jonathan Douglas Finn, Amy Madison Rhoden Smith, Seth C. Alexander
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Abstract
Compositions and methods for gene editing. In some embodiments, a polynucleotide encoding Cas9 is provided that can provide one or more of improved editing efficiency, reduced immunogenicity, or other benefits.
Claims26
An mRNA comprising an open reading frame encoding an RNA-guided DNA-binding agent, wherein the open reading frame has an adenine content ranging from its minimum adenine content to 105% of the minimum adenine content, wherein the mRNA comprises a sequence with at least 98% identity to SEQ ID NO:
- 111, 114, or 117.
An mRNA comprising a sequence with at least 98% identical to SEQ ID NO:
- 111.
An mRNA comprising a sequence with at least 98% identity to SEQ ID NO:
- 111, 114, or 117, and any one or more of the following:
- a. a 5′ cap selected from Cap0, Cap 1, and Cap2;
- b. a 5′ UTR with at least 90% identity to any one of SEQ ID NOs:
- 32, 34, 36, 38, 41, or 75-77;
- and
- c. a 3′ UTR with at least 90% identity to any one of SEQ ID NOs:
- 33, 35, 37, 39, or 40.
An mRNA comprising a sequence identical to SEQ ID NO:
- 111.
Dependencies are inferred from the claim text.