Therapeutic benefit of lentiviral-mediated neonatal intracerebral gene therapy in a mouse model of globoid cell leukodystrophy

Human Molecular Genetics

Volumn 23, Issue 12, 2014, Pages 3250-3268

  Lattanzi, Annalisa ^a,b,d   Salvagno, Camilla ^a   Maderna, Claudio ^a   Benedicenti, Fabrizio ^a   Morena, Francesco ^b   Kulik, Willem ^c   Naldini, Luigi ^a   Montini, Eugenio ^a   Martino, Sabata ^b   Gritti, Angela ^a  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b UNIVERSITY OF PERUGIA   (Italy)

^c UNIVERSITY OF AMSTERDAM   (Netherlands)

^d GÉNÉTHON   (France)

Author keywords

[No Author keywords available]

Indexed keywords

CASPASE 3; GALACTOSYLCERAMIDASE; LENTIVIRUS VECTOR; PSYCHOSINE; BETA GALACTOSIDASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; APOPTOSIS; ARTICLE; ASTROCYTE; BRAIN TISSUE; CELL POPULATION; CELL PROLIFERATION; CONTROLLED STUDY; EXTERNAL CAPSULE; GENE EXPRESSION; GLOBOID CELL LEUKODYSTROPHY; HISTOPATHOLOGY; IMMUNE RESPONSE; LENTIVIRINAE; LIFESPAN; MOUSE; NERVE CELL; NERVOUS SYSTEM DEVELOPMENT; NONHUMAN; OLIGODENDROGLIA; PRIORITY JOURNAL; TARGET CELL; TRANSGENE; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; VIRUS GENOME; ANIMAL; C57BL MOUSE; CENTRAL NERVOUS SYSTEM; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETIC TRANSDUCTION; GENETICS; HEK293 CELL LINE; HUMAN; LEUKODYSTROPHY, GLOBOID CELL; METABOLISM; NEWBORN; PATHOLOGY; THERAPEUTIC USE; VIROLOGY;

ANIMALS; ANIMALS, NEWBORN; BETA-GALACTOSIDASE; CENTRAL NERVOUS SYSTEM; DISEASE MODELS, ANIMAL; EXTERNAL CAPSULE; GENETIC THERAPY; GENETIC VECTORS; HEK293 CELLS; HUMANS; LENTIVIRUS; LEUKODYSTROPHY, GLOBOID CELL; MICE; MICE, INBRED C57BL; TRANSDUCTION, GENETIC;

EID: 84901292902     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddu034     Document Type: Article

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