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

Claim 1:
2. A method of inducing a double-stranded break (DSB) within a TTR gene or modifying the TTR gene, comprising delivering a composition to a cell, wherein the composition comprises a single guide RNA comprising in 5′ to 3′ order, 1) one of the following (a)-(c): a. a guide sequence comprising the sequence of SEQ ID NO: 23; b. a guide sequence that is at least 17, 18, or 19 contiguous nucleotides of the sequence of SEQ ID NO: 23; or c. a guide sequence that is at least 90% identical to the sequence of SEQ ID NO: 23, and (2) the sequence of SEQ ID NO: 125, wherein the sequence of SEQ ID NO: 125 comprises a modification pattern of nucleotides 21-100 of SEQ ID NO: 3 (GUUUUAGAmGmCmUmAmGmAmAmAmUmAmGmCAAGUUAAAAUAAGGCU AGUCCGUUAUCAmAmCmUmUmGmAmAmAmAmAmGmUmGmGmCmAmCmC mGmAmGmUmCmGmGmUmGmCmU*mU*mU*mU), wherein a indicates a phosphorothioate (PS) linkage and a lower case “m” indicates that the nucleotide is 2′-O-Me modified.