C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/11 (2006.01) A61K 48/00 (2006.01) C07H 21/00 (2006.01)
Patent
CA 2560631
The present invention provides methods and compositions for attenuating expression of a target gene in vivo. In general, the method includes administering RNAi constructs (such as small-interfering RNAs (i.e., siRNAs) that are targeted to particular mRNA sequences, or nucleic acid material that can produce siRNAs in a cell), in an amount sufficient to attenuate expression of a target gene by an RNA interference mechanism. In particular, the RANi constructs include one or more modifications to improve serum stability and cellular uptake and to avoid non-specific effect.
La présente invention concerne des méthodes et des compositions destinées à atténuer in vivo l'expression d'un gène cible. En général, la méthode consiste à administrer des constructions d'ARNi (tels que de petits ARN intérférents (c.-à-d. des ARNsi) ciblant des séquences d'ARNm particulières, ou un matériau d'acide nucléique pouvant produire des ARNsi dans une cellule), en quantité suffisante pour atténuer l'expression d'un gène cible par un mécanisme d'interférence de l'ARN. En particulier, les constructions d'ARNi comprennent une ou plusieurs modifications destinées à améliorer la stabilité sérique et l'absorption cellulaire, et à empêcher un effet non spécifique.
Borden Ladner Gervais Llp
California Institute Of Technology
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