Widespread enzymatic correction of CNS tissues by a single intracerebral injection of therapeutic lentiviral vector in leukodystrophy mouse models

Human Molecular Genetics

Volumn 19, Issue 11, 2010, Pages 2208-2227

  Lattanzi, Annalisa ^a   Neri, Margherita ^a,b   Maderna, Claudio ^a   di Girolamo, Ilaria ^c   Martino, Sabata ^c   Orlacchio, Aldo ^c   Amendola, Mario ^a   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

[No Author keywords available]

Indexed keywords

CEREBROSIDE SULFATASE; GALACTOSYLCERAMIDASE; LENTIVIRUS VECTOR; PRIMER DNA;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN STEM; CENTRAL NERVOUS SYSTEM; CEREBELLUM; CEREBROSPINAL FLUID FLOW; CONTROLLED STUDY; ENZYME ACTIVITY; GLOBOID CELL LEUKODYSTROPHY; LEUKODYSTROPHY; METACHROMATIC LEUKODYSTROPHY; MOUSE; NERVE FIBER TRANSPORT; NONHUMAN; NUCLEOTIDE SEQUENCE; PRIORITY JOURNAL; PROTEIN EXPRESSION; PROTEIN LOCALIZATION; SPINAL CORD; TELENCEPHALON; TISSUE DISTRIBUTION; VIRAL GENE DELIVERY SYSTEM; WHITE MATTER; ADMINISTRATION AND DOSAGE; ANIMAL; BIOAVAILABILITY; CONFOCAL MICROSCOPY; ENZYMOLOGY; GEL CHROMATOGRAPHY; GENE THERAPY; GENE VECTOR; GENETICS; IMMUNOHISTOCHEMISTRY; KNOCKOUT MOUSE; LENTIVIRINAE; LEUKODYSTROPHY, GLOBOID CELL; LEUKODYSTROPHY, METACHROMATIC; METABOLISM; PHYSIOLOGY; PROCEDURES; REVERSE TRANSCRIPTION POLYMERASE CHAIN REACTION; WESTERN BLOTTING;

MURINAE; MUS;

ANIMALS; AXONAL TRANSPORT; BIOLOGICAL AVAILABILITY; BLOTTING, WESTERN; CENTRAL NERVOUS SYSTEM; CEREBROSIDE-SULFATASE; CHROMATOGRAPHY, GEL; DNA PRIMERS; GALACTOSYLCERAMIDASE; GENETIC THERAPY; GENETIC VECTORS; IMMUNOHISTOCHEMISTRY; LENTIVIRUS; LEUKODYSTROPHY, GLOBOID CELL; LEUKODYSTROPHY, METACHROMATIC; MICE; MICE, KNOCKOUT; MICROSCOPY, CONFOCAL; REVERSE TRANSCRIPTASE POLYMERASE CHAIN REACTION;

EID: 77953522837     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddq099     Document Type: Article

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