Nucleic acid based modulators of gene expression

C - Chemistry – Metallurgy – 12 – N

Patent

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C12N 15/11 (2006.01) A61K 31/7088 (2006.01) A61P 3/10 (2006.01) A61P 9/00 (2006.01) A61P 25/28 (2006.01) A61P 35/00 (2006.01) C07H 19/00 (2006.01) C07H 19/10 (2006.01) C07H 19/20 (2006.01) C07H 21/00 (2006.01) C12N 5/10 (2006.01) C12N 9/00 (2006.01) C12P 19/30 (2006.01) C12P 19/34 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2403243

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.

L'invention concerne de nouvelles molécules d'acide nucléique qui s'utilisent comme inhibiteurs d'expression génétique, des compositions, et des méthodes d'utilisation desdites molécules.

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