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US 12,227,740 B2Granted
CBLB endonuclease variants, compositions, and methods of use
AssigneeRegeneron Pharmaceuticals, Inc.
InventorsJordan Jarjour, Kyle Havens, Anne-Rachel Krostag
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Abstract
The present disclosure provides improved genome editing compositions and methods for editing a CBLB gene. The disclosure further provides genome edited cells for the prevention, treatment, or amelioration of at least one symptom of, a cancer, an infectious disease, an autoimmune disease, an inflammatory disease, or an immunodeficiency.
Claims11
A method of editing a human CBLB gene in a cell comprising:
- introducing a polynucleotide encoding a polypeptide into the cell, wherein the polynucleotide encodes an I-OnuI homing endonuclease (HE) variant comprising an amino acid sequence selected from the group consisting of SEQ ID NO:
- 6, SEQ ID NO:
- 7, SEQ ID NO:
- 8, SEQ ID NO:
- 9, SEQ ID NO:
- 10, SEQ ID NO:
- 11, and SEQ ID NO:
- 12;
- wherein the I-OnuI HE variant binds and cleaves a target site in the human casitas B-lineage (Cbl) lymphoma proto-oncogene B (CBLB) gene set forth in SEQ ID NO:
- 20,
- wherein the cleaving is at the nucleotides ATTC in SEQ ID NO:
- 20 and creates a double strand break, and
- wherein the break is repaired by non-homologous end joining (NHEJ).
10 more elements
Dependencies are inferred from the claim text.