发明公开
EP1260586A3 Method and reagent for inhibiting the expression of disease related genes
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方法和化合物用于降低基因的与疾病相关的表达
- 专利标题: Method and reagent for inhibiting the expression of disease related genes
- 专利标题(中): 方法和化合物用于降低基因的与疾病相关的表达
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申请号: EP02013004.3申请日: 1995-02-23
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公开(公告)号: EP1260586A3公开(公告)日: 2004-04-28
- 发明人: Stinchcomb, Dan T. , Dudycz, Lech W. , Chowrira, Bharat M. , Grimm, Susan , Direnzo, Anthony , Karpeisky, Alexander , Draper, Kenneth G. , Kisich, Kevin , Matulic-Adamic, Jasenka , McSwiggen, James A. , Modak, Anil , Pavco, Pamela , Beigelman, Leonid , Sullivan, Sean M. , Sweedler, David , Thompson, James D. , Tracz, Danuta , Usman, Nassim , Wincott, Francine E. , Woolf, Tod
- 申请人: RIBOZYME PHARMACEUTICALS, INC.
- 申请人地址: 2950 Wilderness Place Boulder, CO 80301 US
- 专利权人: RIBOZYME PHARMACEUTICALS, INC.
- 当前专利权人: RIBOZYME PHARMACEUTICALS, INC.
- 当前专利权人地址: 2950 Wilderness Place Boulder, CO 80301 US
- 代理机构: Viering, Jentschura & Partner
- 优先权: US201109 19940223; US218934 19940329; US222795 19940404; US224483 19940407; US228041 19940415; US227958 19940415; US245736 19940518; US271280 19940706; US291932 19940815; US291433 19940816; US292620 19940817; US293520 19940819; US300000 19940902; US303039 19940908; US311486 19940923; US311749 19940923; US314397 19940928; US316771 19941003; US319492 19941007; US321993 19941011; US334847 19941104; US337608 19941110; US345516 19941128; US357577 19941216; US363233 19941223; US380734 19950130
- 主分类号: C12N15/11
- IPC分类号: C12N15/11 ; C12N9/00 ; C07H21/00
摘要:
Enzymatic RNA molecules which cleave ICAM-1 mRNA, IL-5 mRNA, rel A mRNA, TNF-α mRNA, RSV mRNA or RSV genomic RNA, or CML associated mRNA, and use of these molecules for the treatment of pathological conditions related to those mRNA-levels; ribonucleosides or nucleotides modified in 2', 3' or 5', methods for their synthesis, purification and deprotection; vectors containing multiple enzymatic nucleic acids, optionally in chimeric form with tRNAs; method for introducing enzymatic nucleic acids into cells by forming a complex with a second nucleic acid, where the complex is capable of taking an R-loop base-paired structure; method for altering a mutant nucleic acid in vivo by hybridization with an oligonucleotide capable of activating ds RNA deaminase, comprising an enzymatic activity or a chemical mutagen. Further are disclosed trans-cleaving or -ligating hairpin ribozymes lacking a substrate RNA moiety, as well as hammerhead ribozymes having an interconnecting loop between base pairs in stem II.
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