Short interfering rna (sirna) analogues

C - Chemistry – Metallurgy – 12 – N

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Details

C12N 15/11 (2006.01) A61K 31/713 (2006.01) A61P 35/00 (2006.01) C07H 21/04 (2006.01)

Patent

CA 2519860

The present invention is directed to novel double-stranded short interfering (siRNA) analogues comprising locked nucleic acid (LNA) monomers. Such compounds induces sequence-specific post-transcriptional gene silencing in many organisms by a process known as RNA interference (RNAi). The compounds disclosed herein has improved properties compared to non-modified siRNAs and may, accordingly, be useful as therapeutic agents, e.g., in the treatment of various cancer forms. More particularly, the present invention is directed to siRNA analogues comprising a sense strand and an antisense strand, wherein each strand comprises 12-35 nucleotides and wherein the siRNA analogues comprise at least one locked nucleic acid (LNA) monomer.

La présente invention concerne des nouveaux analogues de petits ARN interférents (siRNA) en double brin comprenant des monomères d'acide nucléique bloqué (LNA). Ces composés induisent une extinction génique post-transcriptionnelle spécifique de séquence dans une pluralité d'organismes au moyen d'un processus connu sous le nom d'interférence ARN (RNAi). Les composés de l'invention présentent des propriétés améliorées par comparaison avec des siRNA non modifiés, et, par conséquent, peuvent être utiles comme agents thérapeutiques, notamment dans le traitement de diverses formes de cancer. Plus particulièrement, la présente invention concerne des analogues de siRNA comprenant un brin sens et un brin anti-sens, chaque brin comprenant entre 12 et 35 nucléotides, les analogues de siRNA comprenant au moins un monomère d'acide nucléique bloqué (LNA).

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