Methods and compositions for therapeutic use of rna...

A - Human Necessities – 61 – K

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A61K 48/00 (2006.01) A61K 9/00 (2006.01) A61K 9/50 (2006.01) A61K 9/72 (2006.01) A61K 31/7088 (2006.01) A61K 31/7105 (2006.01) A61K 47/30 (2006.01) A61L 27/28 (2006.01) A61M 15/00 (2006.01) C12N 15/87 (2006.01) A61K 9/127 (2006.01) A61K 9/16 (2006.01)

Patent

CA 2465860

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, e.g., in a sequence- dependent, PKR-independent manner. In particular, the subject method can be used to alter the growth, survival or differentiation of cells for therapeutic and cosmetic purposes.

L'invention concerne des procédés et des compositions permettant d'atténuer l'expression d'un gène cible in vivo. En général, ce procédé consiste à administrer des RNAi recombinés (tels que des petits ARN interférants (ou siRNA) ciblés sur des séquences particulières d'ARNm, ou des acides nucléiques capables de produire des siARN dans une cellule), à raison d'une quantité suffisante pour atténuer l'expression d'un gène cible par un mécanisme d'interférence ARN, autrement dit d'une manière dépendante de la séquence, et indépendante de PKR. Le procédé décrit permet en particulier de modifier la croissance, la survie ou la différenciation des cellules à des fins thérapeutiques et cosmétiques.

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