Safe and efficient transduction of the liver after peripheral vein infusion of self-complementary AAV vector results in stable therapeutic expression of human FIX in nonhuman primates

Blood

Volumn 109, Issue 4, 2007, Pages 1414-1421

  Nathwani, Amit C ^a,b,f   Gray, John T ^c   McIntosh, Jenny ^a   Ng, Catherine Y C ^c   Zhou, Junfang ^c   Spence, Yunyu ^c   Cochrane, Melanie ^a   Gray, Elaine ^d   Tuddenham, Edward G D ^a,e   Davidoff, Andrew M ^c  

^a UNIVERSITY COLLEGE LONDON   (United Kingdom)

^b NHS BLOOD AND TRANSPLANT   (United Kingdom)

^c ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

^d NATIONAL INSTITUTE FOR BIOLOGICAL STANDARDS AND CONTROL   (United Kingdom)

^e Thrombosis Unit   (United Kingdom)

^f UNIVERSITY COLLEGE LONDON   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 9;

ADAPTIVE IMMUNITY; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CONTROLLED STUDY; DISEASE SEVERITY; DRUG EFFICACY; DRUG SAFETY; GENE TRANSFER; GENETIC CODE; GENETIC TRANSDUCTION; HEMOPHILIA B; MACACA; MALE; NONHUMAN; PERIPHERAL VEIN; PORTAL VEIN BLOOD FLOW; PRIORITY JOURNAL; SEROTYPE; THERAPY EFFECT; TREATMENT OUTCOME; TREATMENT RESPONSE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS CAPSID; VIRUS STRAIN;

EID: 33846934410     PISSN: 00064971     EISSN: 00064971     Source Type: Journal    
DOI: 10.1182/blood-2006-03-010181     Document Type: Article

References (21)

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