Patent ID: 11965165
Assignee: INTELLIA THERAPEUTICS, INC.
Field: Pharmaceuticals (Chemistry)
Classification: CPC C  A | IPC A  C

Claim 18:
19. A method of inducing a double-stranded break (DSB) within the TTR gene or modifying the TTR gene, comprising administering a composition to a subject in need thereof, wherein the composition comprises (i) a S. pyogenes (Spy) Cas9 or a nucleic acid encoding the SpyCas9 and (ii) a single guide RNA comprising the sequence of SEQ ID NO: 87 (mA*mA*mA*GGCUGCUGAUGACACCUGUUUUAGAmGmCmUmAmGmAmAmAmUm AmGmCAAGUUAAAAUAAGGCUAGUCCGUUAUCAmAmCmUmUmGmAmAmAmAm AmGmUmGmGmCmAmCmCmGmAmGmUmCmGmGmUmGmCmU*mU*mU*mU), wherein a* indicates a phosphorothioate (PS) linkage and a lower case “m” indicates that the nucleotide is 2′-O-Me modified.