AAV5-factor VIII gene transfer in severe hemophilia a

New England Journal of Medicine

Volumn 377, Issue 26, 2017, Pages 2519-2530

  Rangarajan, Savita ^a   Walsh, Liron ^b   Lester, Will ^g   Perry, David ^c   Madan, Bella ^d   Laffan, Michael ^e   Yu, Hua ^g   Vettermann, Christian ^g   Pierce, Glenn F ^h   Wong, Wing Y ^g   Pasi, K John ^f  

^a BASINGSTOKE AND NORTH HAMPSHIRE HOSPITAL   (United Kingdom)

^b UNIVERSITY HOSPITALS BIRMINGHAM NHS FOUNDATION TRUST   (United Kingdom)

^c ADDENBROOKE S HOSPITAL   (United Kingdom)

^d ST THOMAS HOSPITAL   (United Kingdom)

^e IMPERIAL COLLEGE HEALTHCARE NHS TRUST   (United Kingdom)

^f QUEEN MARY UNIVERSITY OF LONDON   (United Kingdom)

^g BIOMARIN PHARMACEUTICAL INC   (United States)

^h NONE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ALANINE AMINOTRANSFERASE; BLOOD CLOTTING FACTOR 8; NEUTRALIZING ANTIBODY; VIRUS VECTOR; VIRUS ANTIBODY; VIRUS DNA;

ADENO ASSOCIATED VIRUS 5; ADULT; ALANINE AMINOTRANSFERASE BLOOD LEVEL; ARTHRALGIA; ARTICLE; BACKACHE; CLINICAL ARTICLE; COHORT ANALYSIS; CONTROLLED CLINICAL TRIAL; CONTROLLED STUDY; COUGHING; DISEASE SEVERITY; DOSE RESPONSE; DRUG DOSE ESCALATION; DRUG EFFICACY; DRUG SAFETY; FATIGUE; GENE TRANSFER; HEMOPHILIA A; HUMAN; IMMUNE RESPONSE; LIMIT OF QUANTITATION; MALE; PHASE 1 CLINICAL TRIAL; PHASE 2 CLINICAL TRIAL; PRIORITY JOURNAL; BLEEDING; BLOOD; CLINICAL TRIAL; DEPENDOPARVOVIRUS; GENE THERAPY; GENE VECTOR; GENETICS; IMMUNOLOGY; INTRAVENOUS DRUG ADMINISTRATION; METABOLISM; MULTICENTER STUDY; PROCEDURES; VIRUS SHEDDING; YOUNG ADULT;

ADULT; ANTIBODIES, VIRAL; DEPENDOVIRUS; DNA, VIRAL; FACTOR VIII; GENETIC THERAPY; GENETIC VECTORS; HEMOPHILIA A; HEMORRHAGE; HUMANS; INFUSIONS, INTRAVENOUS; MALE; VIRUS SHEDDING; YOUNG ADULT;

EID: 85039954002     PISSN: 00284793     EISSN: 15334406     Source Type: Journal    
DOI: 10.1056/NEJMoa1708483     Document Type: Article

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