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METHODS AND COMPOSITIONS FOR MODULATION OF EXPRESSION OF GENES USING COMPONENTS THAT HAVE THE ABILITY TO SELF-ASSEMBLY IN CELLS AND CONDITION RNA-ACTIVITY
专利权人:
СМИТ ЛАРРИ ДЖ. (US)
发明人:
СМИТ Ларри Дж. (US)
申请号:
RU2013151301/10
公开号:
RU2013151301A
申请日:
2012.04.20
申请国别(地区):
RU
年份:
2015
代理人:
摘要:
1. A preparation that inhibits the expression of at least one target ribonucleic acid sequence of interest in an individual cell, comprising: a) a first composition comprising a first nucleic acid strand in a pharmaceutically acceptable in vivo excipient, wherein said strand contains modifications, effectively increasing the stability and activity of the specified first chain in the target tissue in vivo; b) a second composition comprising a second nucleic acid chain in a pharmaceutically acceptable excipient, where seemed chain contains modifications efficiently enhancing stability and activity of said second strand in the target tissue in vivo; wherein said composition does not contain a prodrug or carrier and at least one strand complementary to the target nucleic acid over at least 6 contiguous nucleosides; i) the first and second chains specified in subparagraphs a) and b) are complementary to each other and consist of 13-27 nucleotides and have the ability to form duplex; II) these modifications increase the resistance to nucleases of these chains in vivo; III) these modifications change the Tm of the duplex; and after the sequential introduction of the indicated first and second chains in vivo, these chains show an increased ability to inhibit the expression of the indicated sequence of the target ribonucleic acid in the specified cell in the individual’s body with inhibitory ability achieved when using drugs containing traditional oligonucleotide duplexes of the chains specified in subparagraphs a) and b), or drugs containing one of the chains specified in subparagraph1. Препарат, который ингибирует экспрессию по меньшей мере одной представляющей интерес последовательности рибонуклеиновой кислоты-мишени в клетке индивидуума, содержащий;а) первую композицию, включающую первую цепь нуклеиновой кислоты в фармацевтически приемлемом для введения in vivo наполнителе, где указанная цепь содержит модификации, эффективно повышающие стабильность и активность указанной
来源网站:
中国工程科技知识中心
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