Integration-deficient lentiviral vectors expressing codon-optimized R338L human FIX restore normal hemostasis in hemophilia B mice

Molecular Therapy

Volumn 22, Issue 3, 2014, Pages 567-574

  Suwanmanee, Thipparat ^a   Hu, Genlin ^a   Gui, Tong ^a   Bartholomae, Cynthia C ^b   Kutschera, Ina ^b   Von Kalle, Christof ^b   Schmidt, Manfred ^b   Monahan, Paul E ^a   Kafri, Tal ^a  

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

^b GERMAN CANCER RESEARCH CENTER DKFZ   (Germany)

Author keywords

[No Author keywords available]

Indexed keywords

ARGININE; BLOOD CLOTTING FACTOR 9; COMPLEMENTARY DNA; INTEGRATION DEFICIENT LENTIVIRAL VECTOR; LENTIVIRUS VECTOR; LEUCINE; UNCLASSIFIED DRUG;

AMINO ACID SUBSTITUTION; ANIMAL EXPERIMENT; ANIMAL TISSUE; ARTICLE; BIOSAFETY; CATALYSIS; CELLULAR IMMUNITY; CODON USAGE; DOSE RESPONSE; DRUG EFFICACY; DRUG POTENCY; DRUG SAFETY; GENE REPLACEMENT THERAPY; HEMOPHILIA B; HOMEOSTASIS; HUMORAL IMMUNITY; MOUSE; NONHUMAN; POLYMERASE CHAIN REACTION; PROTEIN SYNTHESIS; VIRAL GENE DELIVERY SYSTEM;

MUS;

ANIMALS; ARGININE; CODON; DISEASE MODELS, ANIMAL; FACTOR IX; GENETIC THERAPY; GENETIC VECTORS; HEMOPHILIA B; HEP G2 CELLS; HUMANS; LENTIVIRUS; LEUCINE; LIVER; MICE; MICE, INBRED C57BL; MICE, KNOCKOUT;

EID: 84895434154     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2013.188     Document Type: Article

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