Directed pseudouridylation of rna
AssigneeThe Regents of the University of California
InventorsEugene Yeo, Kristopher Brannan
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Abstract
Described herein are compositions, systems, methods, and kits utilizing CRISPR-Cas protein fusions comprising a guide nucleotide sequence-programmable RNA binding protein and a RNA pseudouridylation modification protein. The compositions, systems, methods, and kits described herein are useful to modulate RNA pseudouridylation.
Claims30
A fusion protein comprising:
- (i) a guide nucleotide sequence-programmable RNA binding protein;
- and
- (ii) an RNA pseudouridylation modification protein (RPMP).
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A system for modulating RNA pseudouridylation of a target RNA, the system comprising:
- (i) a fusion protein comprising:
- (a) a guide nucleotide sequence-programmable RNA binding protein, and (b) an RNA pseudouridylation modification protein (RPMP);
- and
- (ii) a gRNA;
- or
- (iii) a crRNA and a tracrRNA;
- wherein the gRNA or the crRNA comprises a sequence complementary to a target RNA, and optionally the gRNA or the crRNA comprises a mismatch at a uridine residue.
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A method for treating a disease or condition associated with RNA pseudouridylation of a target RNA in a subject in need thereof, the method comprising administering a fusion protein comprising (i) a guide nucleotide sequence-programmable RNA binding protein, and (ii) an RNA pseudouridylation modification protein (RPMP), a polynucleotide encoding a fusion protein comprising (i) a guide nucleotide sequence-programmable RNA binding protein, and (ii) an RNA pseudouridylation modification protein (RPMP), a vector comprising a polypeptide encoding a fusion protein comprising (i) a guide nucleotide sequence-programmable RNA binding protein, and (ii) an RNA pseudouridylation modification protein (RPMP), a viral particle comprising a vector comprising a polypeptide encoding a fusion protein comprising (i) a guide nucleotide sequence-programmable RNA binding protein, and (ii) an RNA pseudouridylation modification protein (RPMP), or a cell comprising a vector comprising a polypeptide encoding a fusion protein comprising (i) a guide nucleotide sequence-programmable RNA binding protein, and (ii) an RNA pseudouridylation modification protein (RPMP) to the subject, thereby treating the disease or condition associated with RNA pseudouridylation.
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Dependencies are inferred from the claim text.