Potent lna oligonucleotides for the inhibition of hif-1a...

C - Chemistry – Metallurgy – 07 – H

Patent

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C07H 21/04 (2006.01) A61K 31/7115 (2006.01) A61K 31/712 (2006.01) A61K 31/7125 (2006.01) A61P 11/06 (2006.01) A61P 17/06 (2006.01) A61P 19/02 (2006.01) A61P 35/00 (2006.01)

Patent

CA 2587173

The present disclosure relates to an LNA oligonucleotide consisting of a sequence selected from the group consisting of 5'-(T x GxGxcsasasasgscsastscscsTXGX T-3' and 5'-(G X )TXTXascstsgscscststscsTXTX A- 3', wherein capital letters designate a beta-D-oxy-LNA nucleotide analogue, small letters designate a 2-deoxynucleotide, underline designates either a beta-D-oxy-LNA nucleotide analogue or a 2-deoxynucleotide, subscript "s" designates a phosphorothioate link between neighbouring nucleotides/LNA nucleotide analogues, and subscript "x" designates either a phosphorothioate link or a phosphorodiester link between neighbouring nucleotides/LNA nucleotide analogues, and wherein the sequence is optionally extended by up to five 2-deoxynucleotide units. The LNA oligonucleotides are useful for modulating the expression of hypoxia-inducible factor-la (HIF-1a), e.g. in the treatment of cancer diseases, inhibiting angiogenesis, inducing apoptosis, preventing cellular proliferation, or treating an angiogenic disease, e.g. diabetic retinopathy, macular degeneration (ARMD), psoriasis, rheumatoid arthritis and other inflammatory diseases.

L'invention concerne une oligonucléotide LNA comportant une séquence sélectionnée dans le groupe comportant 5'-(T x GxGxcsasasasgscsastscscsTXGX T-3' and 5'-(G X )TXTXascstsgscscststscsTXTX A-3', où les lettres majuscules désignent une substance analogue à la nucléotide bêta-D-oxy-LNA, les lettres minuscules représentent une 2-déoxynucléotide, les lettres soulignées signifient soit une substance analogue à la nucléotide bêta-D-oxy-LNA soit une 2-déoxynucléotide, l'indice "s" désigne un lien phosphorothioate entre des nucléotides et des substances analogues à la nucléotide LNA voisines, l'indice "x" représente un lien phosphorothioate ou un lien phosphorodiester entre des nucléotides et des substances analogues à la nucléotide LNA voisines. La séquence peut éventuellement être étendue jusqu'à cinq unités de 2-déoxynucléotide. Ces oligonucléotides LNA servent à moduler l'expression du facteur-la (HIF-1a) inductible par l'hypoxie, par ex., pour traiter des maladies cancéreuses, inhiber l'angiogénèse, induire l'apoptose, prévenir la prolifération cellulaire ou traiter une maladie angiogénique telle que la rétinopathie diabétique, la dégénération maculaire (ARMD), le psoriasis, la polyarthrite rhumatoïde ou d'autres maladies inflammatoires.

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