Strategies For Knock-Ins At APLP2 Safe Harbor Sites
AssigneeEmendoBio Inc.
InventorsRafi EMMANUEL, Michal GOLAN MASHIACH
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Abstract
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-159641 and compositions, methods, and uses thereof. Further, wherein a method for modifying in a cell at least one allele of the Amyloid Beta Precursor Like Protein 2 (APLP2) gene is disclosed.
Claims59
A method for modifying in a cell at least one allele of the Amyloid Beta Precursor Like Protein 2 (APLP2) gene, the method comprising
- introducing to the cell a composition comprising:
- at least one CRISPR nuclease, or a polynucleotide molecule encoding the CRISPR nuclease;
- and
- an RNA molecule comprising a guide sequence portion having 17-50 nucleotides, or a nucleotide sequence encoding the same,
- wherein a complex of the CRISPR nuclease and the RNA molecule affects a double strand break in at least one allele of the APLP2 gene.
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A composition comprising an RNA molecule comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs:
- 1-159641.
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Dependencies are inferred from the claim text.