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CONJUGATE (OPTIONS), METHOD FOR TREATING DISEASES OF THE NERVOUS SYSTEM (OPTIONS), METHOD FOR VISUALIZING A CELL, DIAGNOSTIC MEANS, AMIDIC COMPOUNDS (OPTIONS), METHOD FOR OBTAINING A CONDITION
专利权人:
НЛИФЕ ТЕРАПЕУТИКС; С.Л. (ES)
发明人:
АЛЬВАРАДО УРБИНА Габриэль (CA),АРТИГАС ПЕРЕС Франсеск (ES),БОРТОЛОССИ БИАССОНИ Аналия (ES),ВИЛА БОВЕР Микель (ES),МОНТЕФЕЛЬТРО Андрес Пабло (ES)
申请号:
RU2012149042/15
公开号:
RU2012149042A
申请日:
2011.04.19
申请国别(地区):
RU
年份:
2014
代理人:
摘要:
1. A conjugate comprising at least one selectivity agent that specifically binds to at least one neurotransmitter carrier and at least one nucleic acid configured to specifically bind to a target molecule expressed in a cell in which a neurotransmitter carrier is expressed. 2. The conjugate according to claim 1, wherein the nucleic acid is selected from the group consisting of a double stranded RNA interfering oligonucleotide, antisense oligonucleotide, gapmer, PNA, ZNA, ribozyme and aptamer. The conjugate according to claim 1, wherein the binding of the nucleic acid to the target molecule is characterized by inhibition of the activity of the target molecule. The conjugate according to claim 1, wherein the selectivity agent is conjugated to the 5'-end of the oligonucleotide. The conjugate of claim 4, wherein the selectivity agent and the oligonucleotide are linked via a linking group. The conjugate according to claim 5, which contains a second oligonucleotide complementary to the first oligonucleotide. The conjugate of claim 2, wherein the interfering RNA is siRNA. The conjugate according to claim 6, which contains a protective group attached to the 5'-end of the second oligonucleotide. The conjugate of claim 8, in which the protective group is characterized by a structure in which: M represents H or a lipid particle, A and B are monomeric units independently selected from the group consisting of monosaccharide and (C — C) alkylene glycol, a and b are these are integers ranging from 0 to 50, s is an integer ranging from 0 to 30, L1, L2 and L3 are binding compounds independently selected from the group consisting of phosphodiester, phosphothioate, carbamate, methylphosphonate, guanidinium, sulfamate, sulfamide1. Конъюгат, включающий по меньшей мере один агент селективности, специфично связывающийся с по меньшей мере одним переносчиком нейромедиатора, и по меньшей мере одну нуклеиновую кислоту, выполненную с возможностью специфично связываться с молекулой-мишенью, экс
来源网站:
中国工程科技知识中心
来源网址:
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