公开(公告)号：EP0746614A1

公开(公告)日：1996-12-11

申请号：EP95909920.0

申请日：1995-02-23

申请人： RIBOZYME PHARMACEUTICALS, INC.

发明人： STINCHCOMB, Dan T. ,  CHOWRIRA, Bharat ,  DIRENZO, Anthony ,  DRAPER, Kenneth G. ,  DUDYCZ, Lech W. ,  GRIMM, Susan ,  KARPEISKY, Alexander ,  KISICH, Kevin ,  MATULIC-ADAMIC, Jasenka ,  McSWIGGEN, James A. ,  KODAK, Anil ,  PAVCO, Pamela ,  BEIGELMAN, Leonid ,  SULLIVAN, Sean M. ,  SWEEDLER, David ,  THOMPSON, James D. ,  TRACZ, Danuta ,  USMAN, Nassim ,  WINCOTT, Francine E. ,  WOOLF, Tod

IPC分类号： C12N15 ,  A61K38 ,  A61K48

CPC分类号： C12N15/113 ,  A61K38/00 ,  A61K48/00 ,  C12N15/101 ,  C12N15/1131 ,  C12N15/1136 ,  C12N15/1138 ,  C12N15/85 ,  C12N15/86 ,  C12N2310/111 ,  C12N2310/121 ,  C12N2310/122 ,  C12N2310/123 ,  C12N2310/1241 ,  C12N2310/126 ,  C12N2310/127 ,  C12N2310/32 ,  C12N2310/322 ,  C12N2310/333 ,  C12N2310/334 ,  C12N2310/335 ,  C12N2310/336 ,  C12N2310/3513 ,  C12N2310/3523 ,  C12N2310/3527 ,  C12N2310/3533 ,  C12N2310/3535 ,  C12N2760/18511 ,  C12N2830/38

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 dsRNA 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.