Serpinc1 iRNA compositions and methods of use thereof

The invention relates to iRNA, e.g., double- stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia. 本发明...

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Hauptverfasser: BETTENCOURT BRIAN, MAIER MARTIN, IVANKA TOUDJARSKA, FOSTER DONALD, MILSTEIN STUART, ALFICA SEHGAL, KUCHIMANCHI SATYANARAYANA, AKIN AKINC, RADZHIV KALLANTKHOTTATKHIL G, MANOKHARAN MUTKHIAKH, CHARISSE KLAUS
Format: Patent
Sprache:chi ; eng
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Zusammenfassung:The invention relates to iRNA, e.g., double- stranded ribonucleic acid (dsRNA), compositions targeting the Serpinc1 gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpinc1 and methods of treating subjects having a bleeding disorder, such as a hemophilia. 本发明涉及靶向Serpinc1基因的iRNA例如双链核糖核酸(dsRNA)组合物,和使用这类iRNA例如dsRNA组合物来抑制Serpinc1表达的方法以及治疗患有出血性障碍例如血友病的受试者的方法。