Intraosseous delivery of lentiviral vectors targeting factor VIII expression in platelets corrects murine hemophilia A

Molecular Therapy

Volumn 23, Issue 4, 2015, Pages 617-626

  Wang, Xuefeng ^a   Shin, Simon C ^a   Chiang, Andy F J ^a   Khan, Iram ^a   Pan, Dao ^b   Rawlings, David J ^a,c   Miao, Carol H ^a,c  

^a SEATTLE CHILDREN S RESEARCH INSTITUTE   (United States)

^b CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^c UNIVERSITY OF WASHINGTON SCHOOL OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; CD45 ANTIGEN; COMPLEMENTARY DNA; ELONGATION FACTOR 1ALPHA; GLYCOPROTEIN IB ALPHA; GLYCOPROTEIN P 15095; GREEN FLUORESCENT PROTEIN; LENTIVIRUS VECTOR; STEM CELL ANTIGEN 1; STEM CELL FACTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY TITER; ARTICLE; BLOOD CLOTTING; BONE MARROW CELL; CELL DIFFERENTIATION; CONTROLLED STUDY; ENZYME LINKED IMMUNOSORBENT ASSAY; EX VIVO GENE TRANSFER; FLOW CYTOMETRY; GENE DOSAGE; GENE EXPRESSION; GENE TARGETING; HEMATOPOIETIC STEM CELL; HEMOPHILIA A; HFVIII GENE; HUMAN; HUMAN CELL; IMMUNE RESPONSE; IN VIVO STUDY; MEGAKARYOCYTE; MOLECULAR DYNAMICS; MOUSE; NONHUMAN; OUTCOME ASSESSMENT; PARTIAL THROMBOPLASTIN TIME; PHENOTYPE; PROMOTER REGION; PROTEIN EXPRESSION; THROMBOCYTE; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; WILD TYPE; ADMINISTRATION AND DOSAGE; ANIMAL; BLOOD; CELL LINE; GENE VECTOR; GENETICS; INTRAOSSEOUS DRUG ADMINISTRATION; LENTIVIRINAE; METABOLISM;

ANIMALIA; MURINAE;

ANIMALS; BLOOD PLATELETS; CELL LINE; FACTOR VIII; GENETIC VECTORS; GREEN FLUORESCENT PROTEINS; HEMOPHILIA A; HUMANS; INFUSIONS, INTRAOSSEOUS; LENTIVIRUS; MICE;

EID: 84926973426     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2015.20     Document Type: Article

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