AAV-mediated gene delivery in adult GM1-gangliosidosis mice corrects lysosomal storage in CNS and improves survival

PLoS ONE

Volumn 5, Issue 10, 2010, Pages

  Baek, Rena C ^a   Boekman, Marike L D ^b,c   Leroy, Stanley G ^b   Tierney, Laryssa A ^b   Sandberg, Michael A ^d   D'Azzo, Alessandra ^e   Seyfried, Thomas N ^a   Sena Esteves, Miguel ^b  

^a BOSTON COLLEGE   (United States)

^b MASSACHUSETTS GENERAL HOSPITAL   (United States)

^c UNIVERSITY MEDICAL CENTER UTRECHT   (Netherlands)

^d MASSACHUSETTS EYE AND EAR INFIRMARY   (United States)

^e ST JUDE CHILDREN S RESEARCH HOSPITAL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE; GLYCOSPHINGOLIPID; PARVOVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CENTRAL NERVOUS SYSTEM; CEREBELLUM NUCLEUS; CONTROLLED STUDY; DRUG ADMINISTRATION ROUTE; DRUG DISTRIBUTION; DRUG EFFICACY; ENZYME ACTIVITY; ENZYME LOCALIZATION; GM1 GANGLIOSIDOSIS; INTRATHALAMIC DRUG ADMINISTRATION; MOTOR PERFORMANCE; MOUSE; NEUROCHEMISTRY; NONHUMAN; OUTCOME ASSESSMENT; SPINAL CORD; SURVIVAL TIME; VIRAL GENE DELIVERY SYSTEM; ADENO ASSOCIATED VIRUS; ANIMAL; EVOKED VISUAL RESPONSE; GENE VECTOR; GENETIC TRANSFECTION; GENETICS; POLYMERASE CHAIN REACTION; ROTAROD TEST; SURVIVAL; THIN LAYER CHROMATOGRAPHY;

MUS;

ANIMALS; CHROMATOGRAPHY, THIN LAYER; DEPENDOVIRUS; EVOKED POTENTIALS, VISUAL; GANGLIOSIDOSIS, GM1; GENETIC VECTORS; MICE; POLYMERASE CHAIN REACTION; ROTAROD PERFORMANCE TEST; SURVIVAL ANALYSIS; TRANSFECTION;

EID: 78149463784     PISSN: None     EISSN: 19326203     Source Type: Journal    
DOI: 10.1371/journal.pone.0013468     Document Type: Article

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