RNAI CONSTRUCTS FOR INHIBITING TTR EXPRESSION AND METHODS OF USE THEREOF

The disclosure relates to RNAi constructs, such as siRNA, for reducing expression of the TTR gene. Methods of using such RNAi constructs to treat or prevent cardiac disease, such as transthyretin-associated cardiomyopathy (ATTR-CM), are also described. L'invention concerne des constructions d&#...

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Hauptverfasser: MURRAY, Justin K, MEADE, Bryan, RULIFSON, Ingrid, LONG, Jason C
Format: Patent
Sprache:eng ; fre
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Zusammenfassung:The disclosure relates to RNAi constructs, such as siRNA, for reducing expression of the TTR gene. Methods of using such RNAi constructs to treat or prevent cardiac disease, such as transthyretin-associated cardiomyopathy (ATTR-CM), are also described. L'invention concerne des constructions d'interférence ARN, telles que le petit ARN interférent, pour réduire l'expression du gène TTR. L'invention concerne également des méthodes d'utilisation de telles constructions d'interférence ARN pour traiter ou prévenir une maladie cardiaque, telle que l'amylose à transthyrétine avec cardiomyopathie (ATTR-CM).