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US 11,884,925 B2Granted
Synthetic immunomodulation with a CRISPR super-repressor in vivo
AssigneeArizona Board of Regents on behalf of Arizona State University
InventorsSamira Kiani, Mo Reza Ebrahimkhani, Farzaneh Moghadam
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Abstract
Provided herein are CRISPR-based synthetic repression systems as well as methods and compositions using the synthetic repression systems to treat septicemia, an adverse immune response in a subject and Waldenström macroglobulinemia.
Claims11
A synthetic repression system for repressing myeloid differentiation primary response 88 (MyD88) expression in vivo comprising:
- (a) a nucleotide sequence encoding a guide RNA (gRNA) comprising
- (i) a nucleic acid encoding a guide sequence comprising a sequence complementary to a portion of MyD88 and
- (ii) an MS2 aptamer target site specific for an RNA binding bacteriophage MS2 coat protein;
- (b) a nucleotide sequence encoding the RNA binding bacteriophage MS2 coat protein fused to a repression domain and comprising a sequence selected from the group consisting of SEQ ID NOs:
- 55, 59, 61, and 63;
- and
- (c) a nucleotide sequence encoding a multifunctional Cas nuclease;
- wherein the nucleotide sequences are packaged in at least one vector for viral delivery.
5 more elements
Dependencies are inferred from the claim text.