摘要 |
Nucleic acid molecules (antisenses or ribozymes) useful as inhibitors of gene expression, especially of HER2, BACE, TERT, PTP-1B, MetAP-2, HBV, phospholamban, presenilin-2 and PKC-alpha. The nucleic acid molecules can be modified in various ways on the sugar and/or base moieties and/or on the phosphate backbone. They are used in pharmaceutical formulations for the treatment of diseases involving increased expression of the target genes. Also disclosed is a method for the synthesis of a modified pyrimidine nucleotide triphosphate and its incorporation into an oligonucleotide. |
申请人 |
RIBOZYME PHARMACEUTICALS, INC.;MCSWIGGEN, JAMES;USMAN, NASSIM;BLATT, LAWRENCE;BEIGELMAN, LEONID;BURGIN, ALEX;KARPEISKY, ALEXANDER;MATULIC-ADAMIC, JASENKA;SWEEDLER, DAVID;DRAPER, KENNETH;CHOWRIRA, BHARAT;STINCHCOMB, DAN;BEAUDRY, AMBER;ZINNEN, SHAWN;LUGWIG, JANOS;SPROAT, BRIAN, S. |
发明人 |
MCSWIGGEN, JAMES;USMAN, NASSIM;BLATT, LAWRENCE;BEIGELMAN, LEONID;BURGIN, ALEX;KARPEISKY, ALEXANDER;MATULIC-ADAMIC, JASENKA;SWEEDLER, DAVID;DRAPER, KENNETH;CHOWRIRA, BHARAT;STINCHCOMB, DAN;BEAUDRY, AMBER;ZINNEN, SHAWN;LUGWIG, JANOS;SPROAT, BRIAN, S. |