Correction of metachromatic leukodystrophy in the mouse model by transplantation of genetically modified hematopoietic stem cells

Journal of Clinical Investigation

Volumn 113, Issue 8, 2004, Pages 1118-1129

  Biffi, Alessandra ^a   De Palma, Michele ^a   Quattrini, Angelo ^b   Del Carro, Ubaldo ^b   Amadio, Stefano ^b   Visigalli, Ilaria ^a   Sessa, Maria ^b   Fasano, Stefania ^b   Brambilla, Riccardo ^b   Marchesini, Sergio ^c   Bordignon, Claudio ^a,d   Naldini, Luigi ^a,d  

^a SAN RAFFAELE SCIENTIFIC INSTITUTE   (Italy)

^b SAN RAFFAELE HOSPITAL   (Italy)

^c UNIVERSITY OF BRESCIA   (Italy)

^d VITA SALUTE SAN RAFFAELE UNIVERSITY   (Italy)

Author keywords

[No Author keywords available]

Indexed keywords

ARYLSULFATASE; CEREBROSIDE SULFATASE; LENTIVIRUS VECTOR;

ANIMAL MODEL; ARTICLE; ARYLSULFATASE A GENE; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DEGENERATIVE DISEASE; DNA MODIFICATION; ENZYME ACTIVITY; FEMALE; GENE; GENE DELIVERY SYSTEM; GENE THERAPY; GENE TRANSFER; HEMATOPOIETIC STEM CELL TRANSPLANTATION; IN VIVO STUDY; MALE; METACHROMATIC LEUKODYSTROPHY; MICROGLIA; MOUSE; NERVOUS SYSTEM; NONHUMAN; PERIPHERAL NERVOUS SYSTEM; PRIORITY JOURNAL; PROGENY; PROTEIN EXPRESSION;

ANIMALS; CELL DIFFERENTIATION; CELL MOVEMENT; DISEASE MODELS, ANIMAL; GENE THERAPY; HEMATOPOIETIC STEM CELL TRANSPLANTATION; LENTIVIRUS; LEUKODYSTROPHY, METACHROMATIC; MICE; MICE, INBRED C57BL; MOTOR ACTIVITY; NERVOUS SYSTEM;

EID: 11144354757     PISSN: 00219738     EISSN: None     Source Type: Journal    
DOI: 10.1172/JCI200419205     Document Type: Article

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