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COMPOUNDS OF TWO-STAIN RNAA TO RHOA AND THEIR APPLICATION

机译:两步RNA到罗汉果的化合物及其应用

摘要

1. A double-stranded nucleic acid molecule for reducing RHOA gene expression containing a coding thread and an antisense strand, said coding thread and an antisense strand selected from an oligonucleotide designated RHOA_48 (SEQ ID NOS: 79 and 113), RHOA_48u (SEQ ID Nos: 80 and 114 ), RHOA_50 (SEQ ID No: 82 and 116) or RHOA_58 (SEQ ID No: 143 and 157), or a pharmaceutically acceptable salt of the indicated molecule. 2. The double-stranded nucleic acid molecule of claim 1, designated RHOA_48, wherein the sequence of said antisense strand contains the sequence 5 'UGUAGCAAGAUGACUUCUG 3' (SEQ ID NO: 113), and the sequence of said coding strand contains the sequence 5 'CAGAAGUCAUCUUGCUACA 3' (SEQ ID no: 79), or a pharmaceutically acceptable salt of said molecule. 3. The double-stranded nucleic acid molecule according to claim 2, which has a structure in which each sign "|" denotes base pairing; in which each of A, C, G and U independently represents an unmodified or modified ribonucleotide or non-standard group; in which each of Z and Z 'may independently be present or absent, but if present independently is 1-5 consecutive nucleotides or groups other than nucleotides or a combination of nucleotides and groups other than nucleotides covalently attached to the 3'-end of the chain in which they are present; in which z "may or may not be present, but if present, is a" capping "group covalently attached to the 5'-end of the sense strand; furthermore, said double-stranded nucleic acid molecule reduces RHOA gene expression; or a pharmaceutically acceptable salt of said molecule. 4. Molecule for
机译:1.一种用于减少RHOA基因表达的双链核酸分子,其包含编码线和反义链,所述编码线和反义链选自命名为RHOA_48(SEQ ID NOS:79和113),RHOA_48u(SEQ ID NO:1)的寡核苷酸。 Nos:80和114),RHOA_50(SEQ ID No:82和116)或RHOA_58(SEQ ID No:143和157)或所示分子的药学上可接受的盐。 2.权利要求1的双链核酸分子,命名为RHOA_48,其中所述反义链的序列包含序列5'UGUAGCAAGAUGACUUCUG 3'(SEQ ID NO:113),并且所述编码链的序列包含序列5。 “ CAGAAGUCAUCUUGCUACA 3”(SEQ ID NO:79),或所述分子的药学上可接受的盐。 3.根据权利要求2的双链核酸分子,其具有其中每个符号“ |”的结构。表示碱基配对;其中A,C,G和U各自独立地表示未修饰或修饰的核糖核苷酸或非标准基团;其中Z和Z'中的每一个可以独立地存在或不存在,但是如果独立地存在,则是1-5个连续的核苷酸或除核苷酸外的基团,或核苷酸和除核苷酸外的基团的组合共价附于其3'端。它们所在的链条;其中z“可能存在或可能不存在,但如果存在,是共价连接至有义链5'端的“封端”基团;此外,所述双链核酸分子降低RHOA基因表达;或该分子的可药用盐4.分子

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