Gene therapy for treating hemophilia A

Compositions and regimens useful in treating hemophilia A are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expressionof a human Factor VIII. 提供了可用于治疗血友病A的组合物和方案。所述组合物包括具有驱动人因子VIII表达的转甲状腺素蛋白增强子和启动子的重组腺相关病毒(rAAV)。...

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Hauptverfasser:	SIDRANE JENNY AGNES, WANG LILI, WILSON JAMES M
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 ORGANIC CHEMISTRY PEPTIDES PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS SPIRITS VINEGAR WINE
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Beschreibung
Zusammenfassung:	Compositions and regimens useful in treating hemophilia A are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expressionof a human Factor VIII. 提供了可用于治疗血友病A的组合物和方案。所述组合物包括具有驱动人因子VIII表达的转甲状腺素蛋白增强子和启动子的重组腺相关病毒(rAAV)。