Methods and compositions for rnai mediated inhibition of...

C - Chemistry – Metallurgy – 07 – H

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C07H 21/02 (2006.01) A01K 67/00 (2006.01) A61K 31/70 (2006.01) C07H 21/04 (2006.01)

Patent

CA 2454183

Methods and compositions are provided for modulating, e.g., reducing, coding sequence expression in mammals. In the subject methods, an effective amount of an RNAi agent, e.g., an interfering ribonucleic acid (such as an siRNA or shRNA) or a transcription template thereof, e.g., a DNA encoding an shRNA, is administered to a non-embryonic mammal, e.g., via a hydrodynamic administration protocol. Also provided are RNAi agent pharmaceutical preparations for use in the subject methods. The subject methods and compositions find use in a variety of different applications, including academic and therapeutic applications.

L'invention concerne des méthodes et des compositions destinées à moduler, et notamment à réduire, l'expression de séquences codantes chez des mammifères. Ces méthodes consistent à administrer une dose efficace d'un agent ARNi, tel qu'un acide ribonucléique interférant (par exemple, un siARN ou un shARN), ou d'une matrice de transcription correspondante, telle qu'un ADN codant pour un shARN, à un mammifère non embryonnaire, notamment au moyen d'un protocole d'administration hydrodynamique. L'invention concerne également des préparations pharmaceutiques d'agent ARNi utilisées dans les méthodes de l'invention. Ces méthodes et ces compositions trouvent une utilité dans une pluralité d'applications différentes, et notamment dans des applications expérimentales et thérapeutiques.

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