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US 11,028,388 B2Granted
CRISPR/Cas-related methods and compositions for treating Usher syndrome and retinitis pigmentosa
AssigneeEditas Medicine, Inc.
InventorsMorgan L. Maeder, David A. Bumcrot
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Abstract
CRISPR/Cas-related compositions and methods for treatment of Usher Syndrome and/or Retinitis Pigmentosa are disclosed herein.
Claims22
A method of altering a cell comprising contacting the cell with:
- (a) a first gRNA comprising a first targeting domain which is complementary with a first target domain from the USH2A gene, wherein the first targeting domain is configured to provide a first cleavage event selected from a first double strand break and a first single strand break in a region of the USH2A gene which is complementary to a sequence that is the same as, or differs by no more than 3 nucleotides from, a nucleic acid sequence selected from the group consisting of SEQ ID NO:635, SEQ ID NO:392, SEQ ID NO:393, SEQ ID NO:394, SEQ ID NO:395, SEQ ID NO:396, SEQ ID NO:397, SEQ ID NO:398, SEQ ID NO:399, SEQ ID NO:400, and SEQ ID NO:401;
- and
- (b) a Cas9 molecule,
- wherein an NHEJ-mediated indel is generated by the first break, resulting in a deletion in the USH2A gene.
1 more element
A method of altering a cell comprising contacting the cell with:
- (a) a first gRNA comprising a first targeting domain which is complementary with a first target domain from the USH2A gene, the first targeting domain comprising a sequence that is the same as, or differs by no more than 3 nucleotides from, a first targeting domain sequence selected from the group consisting of SEQ ID NO:635, SEQ ID NO:392, SEQ ID NO:393, SEQ ID NO:394, SEQ ID NO:395, SEQ ID NO:396, SEQ ID NO:397, SEQ ID NO:398, SEQ ID NO:399, SEQ ID NO:400, and SEQ ID NO:401,
- the first targeting domain configured to provide a cleavage event selected from a first double strand break and a first single strand break, in the USH2A gene;
- and
- (b) a Cas9 molecule.
1 more element
Dependencies are inferred from the claim text.