Pskh-1 ribozymes and uses in disease treatment

C - Chemistry – Metallurgy – 12 – N

Patent

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Details

C12N 15/54 (2006.01) A61K 31/70 (2006.01) C07H 21/02 (2006.01) C12N 9/12 (2006.01) C12N 9/22 (2006.01) C12N 15/11 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2232301

Disclosed is a purified full-length cDNA molecule encoding putative serine kinase enzyme (PSKH-1), and the expression of the cDNA in a recombinant host cell to produce substantially purified PSKH-1, per se. Inactivation of PSKH-1 pre-mRNA or PSKH-1 mRNA halts DNA synthesis and cell division. Also disclosed are ribozymes capable of cleaving PSKH-1 pre-mRNA or mRNA and thus deactivating PSKH-1 translation. Ribozymes of the hammerhead and hairpin motifs, and various compositions containing same, are also disclosed. The ribozymes compositions are used in the treatment of mammalian patients suffering from diseases or medical conditions characterized by abnormal cell proliferation or growth such as cancer and various non-malignant diseases or medical conditions such as autoimmue diseases, allograft rejection and atherosclerosis.

L'invention porte sur une molécule d'ADNc complète purifiée codant une enzyme sérine kinase potentielle (PSKH-1), et sur l'expression de l'ADNc dans une cellule hôte recombinée, afin de produire uniquement PSKH-1 sensiblement purifiée connue en soi. L'inactivation du pré-ARNm ou de l'ARNm ou de l'ARNm de PSKH-1 stoppe la synthèse de l'ADN et la division cellulaire. L'invention porte également sur: des ribozymes capables de couper le pré-ARNm ou l'ARNm de PSKH-1 et de désactiver ainsi la traduction de PSKH-1; des ribozymes à structure en tête de marteau et en épingle à cheveux; et diverses compositions contenant ces ribozymes. Les compositions de ribozymes sont utilisées dans le traitement de mammifères souffrant de maladies ou d'états pathogènes caractérisés par une prolifération ou un développement anormal de cellules, tels que le cancer ou des maladies bénignes; ou des états pathogènes, tels que des maladies auto-immunes, le rejet d'allogreffes ou l'athérosclérose.

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