Patent ID: 11884925
Assignee: ARIZONA BOARD OF REGENTS ON BEHALF OF ARIZONA STATE UNIVERSITY
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 0:
1. 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.