Rna interference for the treatment of heart failure

A - Human Necessities – 61 – K

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Details

A61K 31/7105 (2006.01) A61P 9/04 (2006.01) C12N 15/864 (2006.01) C12Q 1/68 (2006.01) C12Q 1/70 (2006.01) C12N 15/113 (2010.01)

Patent

CA 2711179

The present invention relates to targeted RNAi for the treatment of heart failure by modulating defective cardiac Ca2+ homeostasis via decreasing expression or activity of phospholamban (PLB) using adeno-associated virus (AAV) transfection of cardiomyocytes. Methods for decreasing ventricular arrhythmias, as well as methods for overall improvement of survival from heart failure in subjects are also disclosed. Further, the present invention provides methods which can be used to diagnose susceptibility to treatment by RNAi, and includes pharmaceutical compositions, kits and vectors including an RNAi sequence.

La présente invention porte sur des ARNi ciblés pour le traitement de l'insuffisance cardiaque par modulation de l'homéostasie cardiaque défectueuse du Ca2+ par la diminution de l'expression ou de l'activité du phospholamban (PLB) à l'aide d'une transfection par des virus adéno-associés (AAV) de cardiomyocytes. L'invention porte également sur des procédés de diminution d'arythmies ventriculaires, ainsi que sur des procédés pour l'amélioration globale de la survie à partir d'une insuffisance cardiaque chez des sujets. De plus, la présente invention propose des procédés qui peuvent être utilisés pour diagnostiquer une sensibilité à un traitement par ARNi, et comprend des compositions pharmaceutiques, des coffrets et des vecteurs comprenant une séquence d'ARNi.

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