GENE THERAPY FOR TREATING WILSON'S DISEASE

Compositions and regimens useful in treating Wilson's Disease are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human ATP7B. 提供了可用于治疗威尔逊病的组合物和方案。所述组合物包含具有驱动人类ATP7B表达的甲状腺素运载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, WILSON JAMES, GOVINDASAMY LAKSHMANAN
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 Wilson's Disease are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human ATP7B. 提供了可用于治疗威尔逊病的组合物和方案。所述组合物包含具有驱动人类ATP7B表达的甲状腺素运载蛋白增强子和启动子的重组腺相关病毒(rAAV)。