Therapeutic levels of factor IX expression using a muscle-specific promoter and adeno-associated virus serotype 1 vector

Human Gene Therapy

Volumn 15, Issue 8, 2004, Pages 783-792

  Liu, Yi Lin ^a   Mingozzi, Federico ^a   Rodriguéz Colôn, Sol M ^a   Joseph, Sasha ^a   Dobrzynski, Eric ^a   Suzuki, Takashi ^a   High, Katherine A ^a,b   Herzog, Roland W ^a,c  

^a HOSPITAL OF THE UNIVERSITY OF PENNSYLVANIA   (United States)

^b HOWARD HUGHES MEDICAL INSTITUTE   (United States)

^c CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; PARVOVIRUS VECTOR; PROTEIN ANTIBODY;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY PRODUCTION; ARTICLE; CONTROLLED STUDY; CYTOMEGALOVIRUS; ENHANCER REGION; GENE DELETION; GENE EXPRESSION; GENE TARGETING; GENE TRANSFER; HEMOPHILIA B; IMMEDIATE EARLY GENE; IN VITRO STUDY; IN VIVO STUDY; MOUSE; MUSCLE; MUSCLE FIBER CULTURE; NONHUMAN; PROMOTER REGION; TISSUE SPECIFICITY; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

ANIMALS; ANTIGENS, VIRAL; DEPENDOVIRUS; FACTOR IX; GENE EXPRESSION; GENE THERAPY; GENETIC VECTORS; HEMOPHILIA B; IMMEDIATE-EARLY PROTEINS; MICE; MUSCLE, SKELETAL; PROMOTER REGIONS (GENETICS); TRANSDUCTION, GENETIC; TRANSGENES;

ADENO-ASSOCIATED VIRUS; ADENO-ASSOCIATED VIRUS 2; ANIMALIA; CUCUMBER MOSAIC VIRUS; CYTOMEGALOVIRUS; MURINAE; PARVOVIRUS;

EID: 4143097968     PISSN: 10430342     EISSN: None     Source Type: Journal    
DOI: 10.1089/1043034041648453     Document Type: Article

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