CPC C12N 15/635 (2013.01) [A61K 48/0008 (2013.01); C07K 14/4703 (2013.01); C12N 9/22 (2013.01); C12N 15/11 (2013.01); C12N 15/111 (2013.01); C12N 15/86 (2013.01); C07K 2319/85 (2013.01); C12N 2310/16 (2013.01); C12N 2310/20 (2017.05); C12N 2310/3519 (2013.01); C12N 2830/005 (2013.01)] | 11 Claims |
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.
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