혈우병 A 치료용 유전자 요법

혈우병 A 치료에 유용한 조성물 및 레지멘이 제공된다. 조성물은 인간 인자 VIII의 발현을 구동시키는 프로모터 및 트랜스티레틴 인핸서를 가진 재조합 아데노-관련 바이러스 (rAAV)를 포함한다. 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...

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Hauptverfasser: SIDRANE JENNY AGNES, WANG LILI, WILSON JAMES M
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creator SIDRANE JENNY AGNES
WANG LILI
WILSON JAMES M
description 혈우병 A 치료에 유용한 조성물 및 레지멘이 제공된다. 조성물은 인간 인자 VIII의 발현을 구동시키는 프로모터 및 트랜스티레틴 인핸서를 가진 재조합 아데노-관련 바이러스 (rAAV)를 포함한다. 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 expression of a human Factor VIII.
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subjects 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
title 혈우병 A 치료용 유전자 요법
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