Biallelic knockout of trac
AssigneeEmendoBio Inc.
InventorsLior Izhar, Rafi Emmanuel
Life of this patent
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Abstract
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-1932 and compositions, methods, and uses thereof.
Claims35
A method for inactivating alleles of the T Cell Receptor Alpha Constant (TRAC) gene in a cell, the method comprising
- introducing to the cell a composition comprising:
- at least one CRISPR nuclease, or a sequence encoding a CRISPR nuclease;
- and
- an RNA molecule comprising a guide sequence portion,
- wherein a complex of the CRISPR nuclease and the RNA molecule affects a double strand break in alleles of the TRAC gene,
- wherein the guide sequence portion of the RNA molecule comprises 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs:
- 1-1932.
A method for inactivating alleles of the T Cell Receptor Alpha Constant (TRAC) gene in a cell, the method comprising
- introducing to the cell a composition comprising:
- at least one CRISPR nuclease, or a sequence encoding a CRISPR nuclease;
- and
- an RNA molecule comprising a guide sequence portion,
- wherein a complex of the CRISPR nuclease and the RNA molecule affects a double strand break in alleles of the TRAC gene,
- wherein the guide sequence portion of the RNA molecule comprises 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs:
- 1-1932 modified to contain 1, 2, 3, 4, or 5 nucleotide mismatches relative to a fully-complementary target sequence of the guide sequence portion.
A composition comprising an RNA molecule which comprises 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs:
- 1-1932.
A composition comprising an RNA molecule which comprises 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOS:
- 1-1932, or any one of SEQ ID NOs:
- 1-1932 modified to contain 1, 2, 3, 4, or 5 nucleotide mismatches relative to a fully-complementary target sequence of the guide sequence portion.
Dependencies are inferred from the claim text.