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US 2026/0308639 A1Published application
Biallelic Knockout of ANGPTL3
AssigneeEmendoBio Inc.
InventorRafi EMMANUEL
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Abstract
RNA molecules comprising a guide sequence portion consisting of 17-50 nucleotides and containing 17-22 nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-20347, and compositions, methods, and uses thereof.
Claims24
A method for inactivating at least one allele of the angiopoietin-like protein 3 (ANGPTL3) gene in a cell, the method comprising
- introducing to the cell a composition comprising:
- at least one CRISPR nuclease, or a nucleotide molecule encoding a CRISPR nuclease;
- and
- an RNA molecule comprising a guide sequence portion, or a DNA molecule encoding the RNA molecule,
- wherein a complex of the CRISPR nuclease and the RNA molecule affects a double strand break in at least one allele of the ANGPTL3 gene,
- wherein the guide sequence portion of the RNA molecule consists of 17-50 contiguous nucleotides.
3 more elements
A composition comprising an RNA molecule comprising a guide sequence portion which consists of 17-50 contiguous nucleotides and comprises 17-22 nucleotides in a sequence set forth in any one of SEQ ID NOs:
- 1-20347, or differs by no more than 3 nucleotides from a sequence set forth in any one of SEQ ID NOs:
- 1-20347.
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Dependencies are inferred from the claim text.