Modified RNAi agents

One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or ne...

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Bibliographische Detailangaben
Hauptverfasser:	MAIER MARTIN, KUCHIMANCHI SATYANARAYANA, RAJEEV KALLANTHOTTATHIL G, MANOHARAN MUTHIAH, CHARISSE KLAUS, ZIMMERMANN TRACY
Format:	Patent
Sprache:	chi ; eng
Schlagworte:	BEER BIOCHEMISTRY CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY HUMAN NECESSITIES HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	One aspect of the present invention relates to double-stranded RNAi (dsRNA) duplex agent capable of inhibiting the expression of a target gene. The dsRNA duplex comprises one or more motifs of three identical modifications on three consecutive nucleotides in one or both strand, particularly at or near the cleavage site of the strand. Other aspects of the invention relates to pharmaceutical compositions comprising the dsRNA agents suitable for therapeutic use, and methods of inhibiting the expression of a target gene by administering the dsRNA agents, e.g., for the treatment of various diseaseconditions. 本发明的个方面涉及能够抑制种靶基因的表达的双链RNAi(dsRNA)双链体试剂。该dsRNA双链体在条或两条链中包括个或多个在三个连续核苷酸上具有三个相同修饰的基序，特别是在该链的裂解位点处或附近。本发明的其他方面涉及包括这些适于治疗用途的dsRNA试剂的药物组合物以及通过给予这些dsRNA试剂来抑制种靶基因的表达的方法，例如用于治疗各种疾病病症。