1 SERPINC1 iRNA COMPOSITIONS AND METHODS OF USE THEREOF

• Main Authors: BETTENCOURT BRIAN, MAIER MARTIN, FOSTER DONALD, AKINC AKIN, MILSTEIN STUART, KUCHIMANCHI SATYANARAYANA, RAJEEV KALLANTHOTTATHIL G, SEHGAL ALFICA, TOUDJARSKA IVANKA, MANOHARAN MUTHIAH, CHARISSE KLAUS
• Format: Patent
• Language: English; Korean
• Published: 25.05.2021
• Subjects: BEER; BIOCHEMISTRY; CHEMISTRY; COMPOSITIONS THEREOF; CULTURE MEDIA; DERIVATIVES THEREOF; ENZYMOLOGY; HUMAN NECESSITIES; HYGIENE; MEDICAL OR VETERINARY SCIENCE; METALLURGY; MICROBIOLOGY; MICROORGANISMS OR ENZYMES; MUTATION OR GENETIC ENGINEERING; NUCLEIC ACIDS; NUCLEOSIDES; NUCLEOTIDES; ORGANIC CHEMISTRY; PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES; PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS; SPIRITS; SUGARS; VINEGAR; WINE

본 발명은 iRNA, 예를 들어, 이중-가닥 리보핵산(dsRNA), Serpinc1 유전자를 표적으로 하는 조성물 및 Serpinc1의 발현을 억제하기 위한 이러한 iRNA, 예를 들어, dsRNA, 조성물의 이용 방법, 및 출혈성 장애, 예를 들어, 혈우병을 갖는 대상체의 치료 방법에 관한 것이다. The invention relates to iRNA, e.g., double-stranded ribonucleic acid (dsRNA), compositions targeting the Serpincl gene, and methods of using such iRNA, e.g., dsRNA, compositions to inhibit expression of Serpincl and methods of treating subjects having a bleeding disorder, such as a hemophilia.