AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice

Blood

Volumn 125, Issue 10, 2015, Pages 1553-1561

  Crudele, Julie M ^a   Finn, Jonathan D ^a   Siner, Joshua I ^a   Martin, Nicholas B ^a   Niemeyer, Glenn P ^b   Zhou, Shangzhen ^a   Mingozzi, Federico ^a   Lothrop, Clinton D ^b   Arruda, Valder R ^a,c  

^a CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^b UNIVERSITY OF ALABAMA AT BIRMINGHAM   (United States)

^c UNIVERSITY OF PENNSYLVANIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9 CONCENTRATE; BLOOD CLOTTING FACTOR 9 INHIBITOR; BLOOD CLOTTING INHIBITOR; FIX R 338L; GRANULOCYTE MACROPHAGE COLONY STIMULATING FACTOR; IMMUNOGLOBULIN G2; INTERLEUKIN 10; INTERLEUKIN 15; INTERLEUKIN 18; INTERLEUKIN 6; PARVOVIRUS VECTOR; RECOMBINANT BLOOD CLOTTING FACTOR 9; TUMOR NECROSIS FACTOR ALPHA; UNCLASSIFIED DRUG; BLOOD CLOTTING FACTOR 9; CYTOKINE; FACTOR IX-PADUA; MUTANT PROTEIN; RECOMBINANT PROTEIN;

ADENO ASSOCIATED VIRUS 8; ADULT; ANAPHYLAXIS; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY PRODUCTION; ARTICLE; BLOOD CLOTTING TIME; CONTROLLED STUDY; DISEASE ERADICATION; DOG; FOLLOW UP; GENE TRANSFER; HEMOPHILIA B; HUMORAL IMMUNITY; IMMUNE RESPONSE; IMMUNOGENICITY; IMMUNOLOGICAL TOLERANCE; LONG TERM CARE; MALE; MOUSE; NEPHROTIC SYNDROME; NONHUMAN; PHENOTYPE; PRIORITY JOURNAL; PROTEIN EXPRESSION; RISK FACTOR; STABLE EXPRESSION; THROMBOGENICITY; VIRAL GENE DELIVERY SYSTEM; ADVERSE EFFECTS; AMINO ACID SUBSTITUTION; ANIMAL; ANTAGONISTS AND INHIBITORS; BLOOD; C57BL MOUSE; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; IMMUNOLOGY; MUTANT MOUSE STRAIN; PROCEDURES; THROMBOSIS; TRANSLATIONAL RESEARCH; VIRUS CAPSID;

AMINO ACID SUBSTITUTION; ANIMALS; CAPSID; CYTOKINES; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DOGS; FACTOR IX; GENE EXPRESSION; GENETIC THERAPY; GENETIC VECTORS; HEMOPHILIA B; HUMANS; MALE; MICE; MICE, INBRED C57BL; MICE, MUTANT STRAINS; MUTANT PROTEINS; RECOMBINANT PROTEINS; THROMBOSIS; TRANSLATIONAL MEDICAL RESEARCH;

EID: 84924997217     PISSN: 00064971     EISSN: 15280020     Source Type: Journal    
DOI: 10.1182/blood-2014-07-588194     Document Type: Article

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