Neural stem cell gene therapy ameliorates pathology and function in a mouse model of globoid cell leukodystrophy

Stem Cells

Volumn 29, Issue 10, 2011, Pages 1559-1571

  Neri, Margherita ^a,b   Ricca, Alessandra ^a,c   Di Girolamo, Ilaria ^c   Alcala' Franco, Beatriz ^a   Cavazzin, Chiara ^a   Orlacchio, Aldo ^c   Martino, Sabata ^c   Naldini, Luigi ^a,b   Gritti, Angela ^a  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

^c UNIVERSITY OF PERUGIA   (Italy)

Author keywords

Gene therapy; Leukodystrophies; Lysosomal storage diseases; Nervous system; Neural stem cells; Stem cell transplantation

Indexed keywords

GALACTOSYLCERAMIDASE;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN TISSUE; CELL ACTIVATION; CELL CULTURE; CELL STRUCTURE; CELL SURVIVAL; CONTROLLED STUDY; ENGRAFTMENT; ENZYME ACTIVITY; GENE THERAPY; GLOBOID CELL LEUKODYSTROPHY; HOST CELL; HUMAN; HUMAN CELL; LIFESPAN; MACROGLIA; MICROGLIA; MOTOR PERFORMANCE; MOUSE; NEURAL STEM CELL; NONHUMAN; PROTEIN EXPRESSION; PROTEIN SECRETION; SPINAL CORD; SURVIVAL RATE; TISSUE PRESERVATION;

ANIMALS; BRAIN; CELL DIFFERENTIATION; CELLS, CULTURED; DISEASE MODELS, ANIMAL; ENZYME ACTIVATION; GALACTOSYLCERAMIDASE; GENE THERAPY; GENETIC VECTORS; HUMANS; LENTIVIRUS; LEUKODYSTROPHY, GLOBOID CELL; MICE; MICE, INBRED C57BL; MICE, NEUROLOGIC MUTANTS; NEURAL STEM CELLS; SPINAL CORD; STEM CELL TRANSPLANTATION; TRANSGENES;

MURINAE; MUS;

EID: 80053259922     PISSN: 10665099     EISSN: None     Source Type: Journal    
DOI: 10.1002/stem.701     Document Type: Article

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