Ultra-small rnas as toll-like receptor-3 antagonists

A - Human Necessities – 61 – K

Patent

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A61K 31/713 (2006.01) A61P 27/02 (2006.01) G01N 33/566 (2006.01) C12N 15/113 (2010.01)

Patent

CA 2716326

Provided are methods and compositions for the treatment or prevention of macular degeneration or other diseases or disorders associated with activation of TLR3. Administration of double- stranded RNAs having a length of 22 nucleotides or less treats or prevents macular degeneration or other diseases or disorders associated with activation of TLR3 due to the ability of the RNAs to bind to but not activate TLR3. Furthermore, all double-stranded RNAs (both targeted and non- targeted) of 22 nu-cleotides or less in length can bind to but not activate TLR3 and thereby treat or prevent such conditions. Also provided is a method for increasing the specificity of a desired siRNA target knockdown, the method comprising administering an amount of a target siRNA sufficient to knockdown a target gene and an amount of a double stranded RNA of 22 nucleotides or less which pre-vents the target siRNA from activating TLR3.

L'invention porte sur des procédés et des compositions pour le traitement ou la prévention de la dégénérescence maculaire ou autres maladies ou troubles associés à l'activation de TLR3. L'administration d'ARN double brin ayant une longueur de 22 nucléotides ou moins traite ou prévient une dégénérescence maculaire ou autres maladies ou troubles associés à l'activation de TLR3 en raison de l'aptitude des ARN à se lier à mais à ne pas activer TLR3. De plus, tous les ARN double brin (à la fois ciblés et non ciblés) de 22 nucléotides ou moins de longueur peuvent se lier à mais ne pas activer TLR3 et par là traiter ou prévenir de tels états. L'invention porte également sur un procédé pour augmenter la spécificité d'une inactivation de la cible d'ARNsi désirée, le procédé comprenant l'administration d'une quantité d'ARNsi cible suffisante pour désactiver un gène cible et une quantité d'un ARN double brin de 22 nucléotides ou moins qui empêche l'ARNsi cible d'activer TLR3.

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