发明名称 |
CHIMERIC ANTISENSE OLIGONUCLEOTIDES AND CELL TRANSFECTING FORMULATIONS THEREOF |
摘要 |
Chimeric oligonucleotide of the formula 5'-W-X<1>-Y-X<2>-Z-3', where W represents a 5'-O-alkyl nucleotide, each of X<1> and X<2> represented a block of seven to twelve phosphodiester-linked 2'-O-alkyl ribonucleotides, Y represents a block of five to twelve phosphorothioate-linked deoxyribonucleotides, and Z represents a blocking group effective to block nuclease activity at the 3' end of the oligonucleotide, are described. These compounds exhibit high resistance to endo- and exonucleases, high sequence specificity, and the ability to activate RNAse H, as evidenced by efficient and long-lasting suppression of target mRNA. The oligonucleotides are preferably transfected into cells in formulations containing a lipid-peptoid conjugate carrier molecule of the formula L-linker-[N(CH2CH2NH2)CH2(C=O)-N(CH2CH2R)CH2(C=O)-N(CH2CH2R)CH2(C=O)]3-NH2, where L is a lipid moiety, including a steroid, and each group R is independently selected from alkyl, aminoalkyl, and aralkyl.
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申请公布号 |
WO0116306(A3) |
申请公布日期 |
2001.05.10 |
申请号 |
WO2000US23290 |
申请日期 |
2000.08.25 |
申请人 |
CHIRON CORPORATION;INNIS, MICHAEL, A.;REINHARD, CHRISTOPH, J.;ZUCKERMANN, RONALD, N. |
发明人 |
INNIS, MICHAEL, A.;REINHARD, CHRISTOPH, J.;ZUCKERMANN, RONALD, N. |
分类号 |
C12N15/09;A61K31/7125;A61K38/00;A61K47/48;A61K48/00;A61P43/00;C07H21/04;C12N15/113;(IPC1-7):C12N15/11;A61K31/712;C07H21/00 |
主分类号 |
C12N15/09 |
代理机构 |
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代理人 |
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主权项 |
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地址 |
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