Employing a gain-of-function factor IX variant R338L to advance the efficacy and safety of hemophilia B human gene therapy: Preclinical evaluation supporting an ongoing adeno-associated virus clinical trial

Human Gene Therapy

Volumn 26, Issue 2, 2015, Pages 69-81

  Monahan, Paul E ^a,b   Sun, Junjiang ^a   Gui, Tong ^a   Hu, Genlin ^a   Hannah, William B ^a   Wichlan, David G ^a   Wu, Zhijian ^a,e   Grieger, Joshua C ^a   Li, Chengwen ^a   Suwanmanee, Thipparat ^a   Stafford, Darrel W ^a   Booth, Carmen J ^c   Samulski, Jade J ^d   Kafri, Tal ^a   McPhee, Scott W J ^d   Samulski, R Jude ^a  

^a UNIVERSITY OF NORTH CAROLINA SCHOOL OF MEDICINE   (United States)

^b UNIVERSITY OF NORTH CAROLINA   (United States)

^c YALE UNIVERSITY SCHOOL OF MEDICINE   (United States)

^d ASKLEPIOS BIOPHARMACEUTICAL INC   (United States)

^e NATIONAL EYE INSTITUTE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 9; BLOOD CLOTTING FACTOR 9 R338L; GUANOSINE PHOSPHATE; NEUTRALIZING ANTIBODY; PARVOVIRUS VECTOR; THROMBIN ANTITHROMBIN COMPLEX; UNCLASSIFIED DRUG; RECOMBINANT PROTEIN;

ADULT; AMINO ACID SUBSTITUTION; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY RESPONSE; ARTICLE; AUTOPSY; BLEEDING; CD8+ T LYMPHOCYTE; CLINICAL TRIAL (TOPIC); CODON; CONTROLLED STUDY; DOSE RESPONSE; DRUG DISTRIBUTION; DRUG EFFICACY; DRUG SAFETY; GAIN OF FUNCTION MUTATION; HEMARTHROSIS; HEMOPHILIA B; HEMOPHILIC ARTHROPATHY; HISTOPATHOLOGY; HUMAN; IMMUNOASSAY; IN VIVO STUDY; INTRON; MALE; MOUSE; NONHUMAN; PROTEIN EXPRESSION; SYNOVITIS; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRAL TROPISM; VIRUS CAPSID; VIRUS NEUTRALIZATION; WILD TYPE; ADMINISTRATION AND DOSAGE; ANIMAL; BLOOD; C57BL MOUSE; CHEMISTRY; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE EXPRESSION; GENE THERAPY; GENE VECTOR; GENETIC ENGINEERING; GENETICS; HEMORRHAGE; IMMUNOLOGY; KNOCKOUT MOUSE; LIVER; METABOLISM; PATHOPHYSIOLOGY; PHARMACOKINETICS; PRECLINICAL STUDY; PROCEDURES; TAIL; TISSUE DISTRIBUTION; VIRION; VIROLOGY;

ADENO-ASSOCIATED VIRUS; ANIMALIA; MIRIDAE; MUS;

ANIMALS; ANTIBODIES, NEUTRALIZING; CAPSID; CLINICAL TRIALS AS TOPIC; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DRUG EVALUATION, PRECLINICAL; FACTOR IX; GENE EXPRESSION; GENETIC ENGINEERING; GENETIC THERAPY; GENETIC VECTORS; HEMOPHILIA B; HEMORRHAGE; HUMANS; LIVER; MALE; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT; RECOMBINANT PROTEINS; TAIL; TISSUE DISTRIBUTION; VIRION;

EID: 84923197177     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2014.106     Document Type: Article

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