A Novel Platform for Immune Tolerance Induction in Hemophilia A Mice

Molecular Therapy

Volumn 25, Issue 8, 2017, Pages 1815-1830

  Merlin, Simone ^a   Cannizzo, Elvira Stefania ^a,f   Borroni, Ester ^a   Bruscaggin, Valentina ^a   Schinco, Piercarla ^b   Tulalamba, Warut ^c,d   Chuah, Marinee K ^c,d   Arruda, Valder R ^e   VandenDriessche, Thierry ^c,d   Prat, Maria ^a   Valente, Guido ^a   Follenzi, Antonia ^a  

^a UNIVERSITY OF EASTERN PIEDMONT   (Italy)

^b CITTÀ DELLA SALUTE E DELLA SCIENZA HOSPITAL   (Italy)

^c FREE UNIVERSITY OF BRUSSELS   (Belgium)

^d UNIVERSITY OF LEUVEN   (Belgium)

^e UNIVERSITY OF PENNSYLVANIA   (United States)

^f UNIVERSITY OF MILAN   (Italy)

Author keywords

gene therapy; hemophilia A; inhibitor titers reversion; targeted FVIII expression; Tregs

Indexed keywords

ADENOVIRUS VECTOR; BLOOD CLOTTING FACTOR 8; BLOOD CLOTTING FACTOR 8 ANTIBODY; CD11B ANTIGEN; GREEN FLUORESCENT PROTEIN; MICRORNA; MICRORNA 122; MICRORNA 126; MIRNA 142 3P; NEUTRALIZING ANTIBODY; RECOMBINANT BLOOD CLOTTING FACTOR 8; UNCLASSIFIED DRUG; VASCULAR ENDOTHELIAL CADHERIN; ALLOANTIBODY;

ADENO ASSOCIATED VIRUS; ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ANTIBODY TITER; ARTICLE; BLOOD CLOTTING TIME; BONE MARROW CELL; CONTROLLED STUDY; DRUG TARGETING; ENDOTHELIUM CELL; FVIII GENE; GENE TRANSFER; GENETIC IMMUNIZATION; HEMATOPOIETIC CELL; HEMOPHILIA A; IMMUNOLOGICAL TOLERANCE; IN VIVO STUDY; LIVER; LIVER CELL; LIVER SINUSOID ENDOTHELIAL CELL; MEMORY T LYMPHOCYTE; MOUSE; NONHUMAN; PROTEIN SECRETION; REGULATORY T LYMPHOCYTE; SPLEEN; TRANSCRIPTION REGULATION; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; 129 MOUSE; ANIMAL; ANTIBODY SPECIFICITY; BAGG ALBINO MOUSE; BLOOD; C57BL MOUSE; DISEASE MODEL; GENE EXPRESSION; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; IMMUNIZATION; IMMUNOLOGY; IMMUNOSUPPRESSIVE TREATMENT; LENTIVIRUS; MALE; METABOLISM; PROCEDURES; PROMOTER REGION; REPORTER GENE;

ANIMALS; CD11B ANTIGEN; DISEASE MODELS, ANIMAL; ENDOTHELIAL CELLS; FACTOR VIII; GENE EXPRESSION; GENES, REPORTER; GENETIC VECTORS; HEMOPHILIA A; IMMUNE TOLERANCE; IMMUNIZATION; IMMUNOSUPPRESSION; ISOANTIBODIES; LENTIVIRUS; MALE; MICE; MICE, 129 STRAIN; MICE, INBRED BALB C; MICE, INBRED C57BL; ORGAN SPECIFICITY; PROMOTER REGIONS, GENETIC; T-LYMPHOCYTES, REGULATORY; TRANSDUCTION, GENETIC; TRANSGENES; WHOLE BLOOD COAGULATION TIME;

EID: 85019953446     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1016/j.ymthe.2017.04.029     Document Type: Article

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