Preclinical Testing of the Safety and Tolerability of Lentiviral Vector-Mediated Above-Normal Alpha-L-Iduronidase Expression in Murine and Human Hematopoietic Cells Using Toxicology and Biodistribution Good Laboratory Practice Studies

Human Gene Therapy

Volumn 27, Issue 10, 2016, Pages 813-829

  Visigalli, Ilaria ^a   Delai, Stefania ^a   Ferro, Francesca ^a   Cecere, Francesca ^a   Vezzoli, Michela ^a   Sanvito, Francesca ^a,b   Chanut, Franck ^c,e   Benedicenti, Fabrizio ^a   Spinozzi, Giulio ^a   Wynn, Rob ^f   Calabria, Andrea ^a   Naldini, Luigi ^a,d   Montini, Eugenio ^a   Cristofori, Patrizia ^a,c   Biffi, Alessandra ^a  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b SAN RAFFAELE HOSPITAL   (Italy)

^c GLAXOSMITHKLINE   (United Kingdom)

^d VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^e SANOFI   (France)

^f ROYAL MANCHESTER CHILDREN S HOSPITAL   (United Kingdom)

Author keywords

[No Author keywords available]

Indexed keywords

COMPLEMENTARY DNA; GENOMIC DNA; LENTIVIRUS VECTOR; LEVO IDURONIDASE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CARCINOGENICITY; CELL DIFFERENTIATION; CELLULAR DISTRIBUTION; CONTROLLED STUDY; DISEASE MARKER; DRUG SAFETY; DRUG TOLERABILITY; ENZYME ACTIVITY; GENE TRANSFER; GOOD LABORATORY PRACTICE; GRAFT RECIPIENT; HEMATOPOIETIC STEM CELL; HISTOPATHOLOGY; HUMAN; HUMAN CELL; HURLER SYNDROME; IN VIVO STUDY; MOUSE; NONHUMAN; PRECLINICAL STUDY; PROGENY; PROTEIN EXPRESSION; STEM CELL GENE THERAPY; TOXICITY TESTING; VIRAL GENE THERAPY; ANIMAL; ENZYMOLOGY; GENE EXPRESSION REGULATION; GENE THERAPY; GENE VECTOR; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; LENTIVIRUS; PROCEDURES;

ANIMALS; GENE EXPRESSION REGULATION, ENZYMOLOGIC; GENE TRANSFER TECHNIQUES; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; IDURONIDASE; LENTIVIRUS; MICE; MUCOPOLYSACCHARIDOSIS I;

EID: 84989244947     PISSN: 10430342     EISSN: 15577422     Source Type: Journal    
DOI: 10.1089/hum.2016.068     Document Type: Article

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