Modified guide RNAs
AssigneeIntellia Therapeutics, Inc.
InventorsAmy Madison Rhoden Smith, David V. Morrissey, Walter Strapps
Life of this patent
Estimating the term…
Abstract
This disclosure relates to modified single and dual guide RNAs having improved in vitro and in vivo activity in gene editing methods.
Claims22
A single guide RNA (sgRNA) comprising the nucleotide sequence mN*mN*mN*NNNNNNNNNNNNNNNNNGUUUUAGAmGmCmUmAmGmAmAmAmUmA mGmCAAGUUAAAAUAAGGCUAGUCCGUUAUCAmAmCmUmUmGmAmAmAmAmAm GmUmGmGmCmAmCmCmGmAmGmUmCmGmGmUmGmCmU*mU*mU*mU, wherein N denotes any one of A, C, G, and U;
- * denotes a phosphorothioate (PS) linkage;
- and mN, mA, mC, mG, and mU respectively denote N, A, C, G, and U modified with 2′-O-methyl (2′-O-Me) (SEQ ID NO:
- 358) of SEQ ID NO:
- 358.
A sgRNA consisting of the nucleotide sequence mN*mN*mN*NNNNNNNNNNNNNNNNNGUUUUAGAmGmCmUmAmGmAmAmAmUmA mGmCAAGUUAAAAUAAGGCUAGUCCGUUAUCAmAmCmUmUmGmAmAmAmAmAm GmUmGmGmCmAmCmCmGmAmGmUmCmGmGmUmGmCmU*mU*mU*mU, wherein N denotes any one of A, C, G, and U;
- * denotes a phosphorothioate (PS) linkage;
- and mN, mA, mC, mG, and mU respectively denote N, A, C, G, and U modified with 2′-O-methyl (2′-O-Me) (SEQ ID NO:
- 358) of SEQ ID NO:
- 358.
A composition comprising:
- (a) a sgRNA comprising the nucleotide sequence mN*mN*mN*NNNNNNNNNNNNNNNNNGUUUUAGAmGmCmUmAmGmAmAmAmUmA mGmCAAGUUAAAAUAAGGCUAGUCCGUUAUCAmAmCmUmUmGmAmAmAmAmAm GmUmGmGmCmAmCmCmGmAmGmUmCmGmGmUmGmCmU*mU*mU*mU, wherein N denotes any one of A, C, G, and U;
- * denotes a phosphorothioate (PS) linkage;
- and mN, mA, mC, mG, and mU respectively denote N, A, C, G, and U modified with 2′-O-methyl (2′-O-Me) (SEQ ID NO:
- 358);
- (b) a mRNA encoding a S. pyogenes Cas9 protein;
- and (c) a lipid nanoparticle (LNP).
A composition comprising:
- (a) a sgRNA consisting of the nucleotide sequence mN*mN*mN*NNNNNNNNNNNNNNNNNGUUUUAGAmGmCmUmAmGmAmAmAmUmA mGmCAAGUUAAAAUAAGGCUAGUCCGUUAUCAmAmCmUmUmGmAmAmAmAmAm GmUmGmGmCmAmCmCmGmAmGmUmCmGmGmUmGmCmU*mU*mU*mU, wherein N denotes any one of A, C, G, and U;
- * denotes a phosphorothioate (PS) linkage;
- and mN, mA, mC, mG, and mU respectively denote N, A, C, G, and U modified with 2′-O-methyl (2′-O-Me) (SEQ ID NO:
- 358);
- (b) a mRNA encoding a S. pyogenes Cas9 protein;
- and (c) a lipid nanoparticle (LNP).
Dependencies are inferred from the claim text.