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 15/63 (2006.01) A61K 48/00 (2006.01) C07H 21/04 (2006.01) C12N 15/11 (2006.01) A61K 38/00 (2006.01)

Patent

CA 2331782

The present invention relates to multi-ribozymes and their use to target RNA in a tissue-specific, target RNA-specific, or pathogen-specific manner for the treatment of cancers, proliferative disease, and bacterial, parasitic and viral infections. More specifically, the present invention relates to the use of virions and viral vectors to package and deliver DNA encoding the multi- ribozymes to a host. The present invention relates to the use of liposomes and lipid-DNA complexes to deliver DNA encoding ribozymes to a host. Most specifically, the invention relates to the use of target specific virions to package and deliver DNA comprising a target specific promoter and encoding a ribozyme(s) directed to the target organism nucleic acids. The present invention further relates to a novel vectors encoding a multi-ribozyme structure with enhanced 5' and/or 3' autocatalytically cleaving ribozymes. The invention further relates to nucleotides encoding a multi-ribozyme comprising one or more ribozyme cassettes which contain one or more trans-acting ribozymes and one or more autocatalytically cleaving ribozyme sequences.

La présente invention concerne des ribozymes multiples et leur utilisation pour cibler l'ARN de manière spécifique aux tissus, à l'ARN cible ou aux pathogènes dans le traitement de cancers, de maladies proliférantes et d'infections bactériennes, parasitaires et virales. Plus spécifiquement, la présente invention concerne l'utilisation de virions et de vecteurs viraux pour conditionner et délivrer un ADN codant les ribozymes multiples à un hôte. La présente invention concerne l'utilisation de liposomes et de complexes lipides-ADN pour délivrer un ADN codant des ribozymes à un hôte. Plus spécifiquement encore, l'invention concerne l'utilisation de virions spécifiques de cibles pour conditionner et délivrer un ADN comprenant un promoteur spécifique de cibles et codant un ribozyme (des ribozymes) dirigé vers les acides nucléiques de l'organisme cible. La présente invention concerne en outre de nouveaux vecteurs codant une structure à ribozymes multiples comprenant des ribozymes à clivage autocatalytique 5' et/ou 3' améliorés. L'invention concerne enfin des nucléotides codant un ribozyme multiple comprenant une ou plusieurs cassettes de ribozymes qui contiennent un ou plusieurs ribozymes trans-activateurs et une ou plusieurs séquences de ribozymes à clivage autocatalytique.

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