Rna interference mediated inhibition of epithelial sodium...

C - Chemistry – Metallurgy – 12 – N

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C12N 15/113 (2010.01) A61K 31/713 (2006.01)

Patent

CA 2744694

The present invention relates to compounds, compositions, and methods for the study, diagnosis, and treatment of traits, diseases and conditions that respond to the modulation of ENaC gene expression and/or activity, and/or modulate a ENaC gene expression pathway. Specifically, the invention relates to double- stranded nucleic acid molecules including small nucleic acid molecules, such as short interfering nucleic acid (siNA), short interfering RNA (siRNA), double-stranded RNA (dsRNA), mi-cro-RNA (miRNA), and short hairpin RNA (shRNA) molecules that are capable of mediating or that mediate RNA interference (RNAi) against ENaC gene expression.

La présente invention concerne des composés, des compositions, et des procédés pour létude, le diagnostic, et le traitement de caractères, maladies et affections qui répondent à la modulation de lexpression et/ou lactivité du gène ENaC, et/ou modulent une voie dexpression du gène ENaC. Spécifiquement, linvention concerne des molécules dacide nucléique bicaténaires comprenant des petites molécules dacide nucléique, telles que des molécules dacide nucléique interférant court (ANsi), dARN interférant court (ARNsi), dARN bicaténaire (ARNdb), de micro-ARN (miARN), et dARN court en épingle à cheveux (ARNsh) qui sont capables de véhiculer ou qui véhiculent lARN interférence (ARNi) contre lexpression du gène ENaC.

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