Tissue-specific and target rna-specific ribozymes

Details Tissue-specific and target rna-specific ribozymes Tissue-specific and target rna-specific ribozymes

C - Chemistry, Metallurgy

12

N

C12N 15/11 (2006.01) A01K 67/027 (2006.01) A61K 31/70 (2006.01) C12N 15/88 (2006.01) C12Q 1/68 (2006.01) A61K 38/00 (2006.01) A61K 48/00 (2006.01)

Patent

CA 2236998

The invention provides tissue-specific and target RNA--specific ribozymes. These ribozymes can be used to destroy target-specific neoplasms and to treat viral infections, among other uses. The ribozymes of the present invention comprise a 5' autocatalytically cleaving ribozyme sequence, a catalytic ribozyme comprising a target RNA-specific binding site and a 3' autocatalytically cleaving ribozyme. The invention also provides nucleic acids which encode the ribozymes of the invention. These nucleic acids can be used to express the ribozymes of the invention at the selected site. Methods of treating disease by administering the ribozymes are provided.

On décrit des ribozymes, spécifiques d'un tissu ou d'un ARN cible, pouvant s'utiliser pour détruire des néoplasmes spécifiques d'une cible et pour traiter des infections virales, entre autres utilisations. Ces ribozymes comprennent une séquence de ribozymes à coupure autocatalytique en 5', un ribozyme catalytique comprenant un site de liaison spécifique d'un ARN cible et un ribozyme à coupure autocatalytique en 3'. On décrit aussi des acides nucléiques qui codent de tels ribozymes et peuvent servir à exprimer les ribozymes décrits sur un site choisi. On décrit enfin des procédés concernant le traitement de maladies et consistant à administrer ces ribozymes.

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