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OLIGORIBONUCLEOTIDES AND WAYS OF THEIR APPLICATION FOR TREATMENT OF ACUTE RENAL FAILURE AND OTHER DISEASES
OLIGORIBONUCLEOTIDES AND WAYS OF THEIR APPLICATION FOR TREATMENT OF ACUTE RENAL FAILURE AND OTHER DISEASES
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机译:寡核糖核苷酸及其在治疗急性肾功能衰竭和其他疾病中的应用方式
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摘要
1. The compound having the structure 5 '(N) -Z3' (antisense chain) 3'Z '- (N') 5 '(sense chain) where each N and N' means ribonucleotide, which may be modified or unmodified at its residue , and (N) and (N ') means an oligomer in which each consecutive N or N' is connected with the subsequent N or N 'covalent bond; where each of x and y is an integer from 19 to 40; where each of Z and Z 'may be present or absent, but if present, means dTdT and is covalently attached at the 3'-end of the chain in which it is present; where sequence (N) contains relative to p53.2 mRNA. The compound according to claim 1, where the sequence (N) contains one or more antisense sequences shown in tables A, B and C.3. A compound according to claim 2, wherein the covalent bond is a phosphodiester bond. The compound according to claim 3, where x = y, preferably, where x = y = 19.5. The compound according to claim 4, where both Z and Z 'are absent. A compound according to claim 4, wherein one of Z or Z ′ is present. The compound according to claim 5, where all the ribonucleotides are unmodified by their sugar residues. A compound according to claim 5, wherein at least one ribonucleotide is modified according to its sugar residue. The compound of claim 8, wherein the modification of the sugar residue comprises a modification at position 2'.10. The compound according to claim 9, wherein the modification at position 2 ′ results in the formation of a part of a molecule selected from the group from amino, fluoro, methoxy, alkoxy and alkyl groups. The compound of claim 10, wherein said portion of the molecule at position 2 ′ is methoxy (2′-O-methyl). The compound of claim 11, wherein the antisymmetric ribonucleotides are modified in both the antisense and sense chains. The compound of claim 12, wherein the rib
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