Sirna-hydrophilic polymer conjugates for intracellular...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/11 (2006.01) C12N 15/87 (2006.01)

Patent

CA 2619533

Disclosed are hybrid conjugates formed by covalently bonding siRNA (small interfering RNA) molecules to hydrophilic polymers for improving stability of the siRNA molecules effective for gene therapy in vivo, and polyelectrolyte complex micelles formed by ionic interactions between the conjugates and multifunctional cationic compounds. The siRNA-hydrophilic polymer conjugates and polyelectrolyte complex micelles derived therefrom can be advantageously used for improving stability of the siRNA molecules in vivo. Consequently, the delivery of siRNA molecules for therapeutic applications into cells can be facilitated, and the siRNA is still active even though a small dose of the siRNA is used.

L'invention concerne des conjugués hybrides formés par liaison covalente de molécules de siARN (petit ARN interférent) à des polymères hydrophiles permettant d'augmenter la stabilité des molécules de siARN, ces conjugués étant utiles pour une thérapie génique in vivo, ainsi que des micelles à complexe polyélectrolytique formées par interactions ioniques entre les conjugués et des composés cationiques multifonctionnels. Les conjugués siARN-polymères hydrophiles et les micelles à complexe polyélectrolytique dérivés de ceux-ci peuvent être utilisés avantageusement pour augmenter la stabilité des molécules de siARN in vivo. Par conséquent, l'administration de molécules de siARN en vue d'applications thérapeutiques dans des cellules peut être facilitée, et le siARN est encore actif même si une petite dose de siARN est utilisée.

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