C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 31/70 (2006.01) A61K 35/12 (2006.01) A61K 35/14 (2006.01) C07D 239/46 (2006.01) C07D 473/00 (2006.01) C07H 19/04 (2006.01) C07H 19/06 (2006.01) C07H 19/10 (2006.01) C07H 19/16 (2006.01) C07H 19/20 (2006.01) C07H 21/00 (2006.01) C07H 21/02 (2006.01) C12N 5/10 (2006.01) C12N 9/22 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2207593
An enzymatic nucleic acid molecule which cleaves RNA associated with development or maintenance of an arthritic condition, induction of graft tolerance or reversal of an immune response. In particular, the ribozyme sequences are directed to an mRNA encoding B7-1, B7-2, B7-3, CD40 and/or stromelysin. Also provided are ribozymes where the uracil in positions 4 and/or 7 are substituted, as well as methods for the synthesis of 2'- alkylnucleotides, 2'-O-alkylthioalkyl, or 2'-alkylthioalkylnucleotides. The application further describes a method for diprotection of RNA with aqueous ethylamine, a method for synthesis of a basic ribonucleoside mimetics, and transcription units comprising an RNA polymerase II promoter, a U6 small nuclear promoter, or an adenovirus VA1 promoter system.
Molécule d'acide nucléique enzymatique effectuant le clivage de l'ARN associé à l'évolution ou à l'existence d'un état arthritique, à l'introduction d'une tolérance aux greffes ou à l'inversion d'une réaction immune. En particulier, les séquences de ribozymes sont dirigées vers un ARNm codant B7-1, B7-3, cd40 et/ou stromelysine. L'invention concerne également des ribozymes où uracil des positions 4 et/ou 7 est substitué, ainsi que des procédés de synthèse de 2'-alkylnucléotides, 2'-O-alkylthioalkyle ou 2'-alkylnucléotides, 2'-O-alkylthioalkyle ou 2'-alkylthioalkylnucléotides. Elle concerne, de plus, un procédé de déprotection de l'ARN par éthylamine aqueux, un procédé de synthèse de produits mimétiques de ribonucléosides de base, ainsi que des unités de transcription composés d'un promoteur de polymérase II d'ARN, d'un promoteur nucléaire réduit de U6 ou d'un système promoteur de VA1 d'adénovirus.
Beigelman Leonid
Burgin Alex
Draper Kenneth
Gustofson John
Jarvis Thale
Ribozyme Pharmaceuticals Inc.
Smart & Biggar
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