Microrna (mirna) and downstream targets for diagnostic and...

C - Chemistry – Metallurgy – 12 – N

Patent

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C12N 15/11 (2006.01) A61K 31/7105 (2006.01) C12Q 1/68 (2006.01)

Patent

CA 2716643

The present invention relates to a promoter region of a microRNA, the use of a microRNA, in particular miR-21, and related elements for the diagnosis and for the manufacture of a medicament for the treatment and/or prevention of fibrosis and/or fibrosis related diseases. Additionally, the invention concerns antisense oligonucleotides against targets of miR-21. A cell deficient for miR-21, the promoter region and targets of miR-21 and a knock- out organism thereof are also encompassed. Finally, the invention is directed to a method for diagnosing fibrosis and/or fibrosis related diseases and to a method for screening a pharmaceutically active compound for the treatment of fibrosis and/or fibrosis related diseases. The present invention further relates to compositions for use in the treatment, amelioration, and/or prevention of fibrosis. In certain embodiments, the compositions modulate the activity of a miRNA for the treatment, amelioration, and/or prevention of fibrosis. In certain embodiments, the compositions inhibit the activity of miR-21 for the treatment, amelioration, and/or prevention of fibrosis.

La présente invention concerne une région promoteur d'un microARN, l'utilisation d'un microARN, en particulier miR-21, et des éléments associés dans le diagnostic et la fabrication d'un médicament pour le traitement et/ou la prévention de la fibrose et/ou de maladies associées à la fibrose. L'invention concerne, de plus,des oligonucléotides antisens utilisés contre des cibles de miR-21. L'invention concerne également une cellule déficiente en miR-21, la région promoteur et des cibles de miR-21 et un organisme knock-out associé. L'invention concerne enfin une méthode de diagnostic de la fibrose et/ou de maladies associées à la fibrose et une méthode de criblage d'un composé pharmaceutiquement actif dans le traitement de la fibrose et/ou de maladies associées à la fibrose. La présente invention concerne également des compositions utilisées dans le traitement, l'amélioration et/ou la prévention de la fibrose. Dans certains modes de réalisation, les compositions modulent l'activité d'un miARN pour le traitement, l'amélioration et/ou la prévention de la fibrose. Dans certains modes de réalisation, les compositions inhibent l'activité de miR-21 pour le traitement, l'amélioration et/ou la prévention de la fibrose.

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