Characterization of adeno-associated viral vector-mediated human factor VIII gene therapy in hemophilia a mice

Human Gene Therapy

Volumn 28, Issue 5, 2017, Pages 392-402

  Greig, Jenny A ^a   Wang, Qiang ^a   Reicherter, Amanda L ^a   Chen, Shu Jen ^a   Hanlon, Alexandra L ^a   Tipper, Christopher H ^b   Clark, K Reed ^b   Wadsworth, Samuel ^b   Wang, Lili ^a   Wilson, James M ^a  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b ULTRAGENYX PHARMACEUTICAL INC   (United States)

Author keywords

AAV; gene therapy; hemophilia A; human coagulation factor VIII

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; BLOOD CLOTTING FACTOR 8; CAPSID PROTEIN;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY PRODUCTION; ARTICLE; CONTROLLED STUDY; ENHANCER REGION; GENETIC VARIABILITY; HEMOPHILIA A; IMMUNOGENICITY; MALE; MOUSE; MOUSE MODEL; NONHUMAN; PROMOTER REGION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS CAPSID; ANIMAL; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE EXPRESSION REGULATION; GENE THERAPY; GENE TRANSFER; GENE VECTOR; GENETICS; HUMAN; IMMUNOLOGICAL TOLERANCE; IMMUNOLOGY; KNOCKOUT MOUSE; LIVER; METABOLISM; THERAPEUTIC USE; TRANSGENE;

ANIMALS; CAPSID PROTEINS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; FACTOR VIII; GENE EXPRESSION REGULATION; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HEMOPHILIA A; HUMANS; IMMUNE TOLERANCE; LIVER; MICE; MICE, KNOCKOUT; PROMOTER REGIONS, GENETIC; TRANSGENES;

EID: 85019240457     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2016.128     Document Type: Article

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