페닐케톤뇨증을 치료하기 위한 유전자 치료

페닐케톤뇨증을 치료하는 데 유용한 조성물 및 섭생이 제공된다. 상기 조성물은 인간 페닐알라닌 수산화효소의 발현을 추진시키는 트란스타이레틴 인핸서 및 촉진자를 갖는 재조합 아데노 연관된 바이러스(rAAV)를 포함한다. Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and pro...

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Bibliographische Detailangaben
Hauptverfasser:	SIDRANE JENNY AGNES, WILSON JAMES M, ASHLEY SCOTT
Format:	Patent
Sprache:	kor
Schlagworte:	AGRICULTURE ANIMAL HUSBANDRY BEER BIOCHEMISTRY CARE OF BIRDS, FISHES, INSECTS CHEMISTRY COMPOSITIONS THEREOF CULTURE MEDIA ENZYMOLOGY FISHING FORESTRY HUMAN NECESSITIES HUNTING HYGIENE MEDICAL OR VETERINARY SCIENCE METALLURGY MICROBIOLOGY MICROORGANISMS OR ENZYMES MUTATION OR GENETIC ENGINEERING NEW BREEDS OF ANIMALS PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES PROPAGATING, PRESERVING OR MAINTAINING MICROORGANISMS REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS ORMEDICINAL PREPARATIONS SPIRITS TRAPPING VINEGAR WINE
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Zusammenfassung:	페닐케톤뇨증을 치료하는 데 유용한 조성물 및 섭생이 제공된다. 상기 조성물은 인간 페닐알라닌 수산화효소의 발현을 추진시키는 트란스타이레틴 인핸서 및 촉진자를 갖는 재조합 아데노 연관된 바이러스(rAAV)를 포함한다. Compositions and regimens useful in treating phenylketonuria are provided. The compositions include recombinant adeno-associated virus (rAAV) with a transthyretin enhancer and promoter driving expression of a human phenylalanine hydroxylase.