Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan

Human Molecular Genetics

Volumn 24, Issue 15, 2015, Pages 4353-4364

  Weismann, Cara M ^a   Ferreira, Jennifer ^a   Keeler, Allison M ^a   Su, Qin ^a   Qui, Linghua ^a   Shaffer, Scott A ^a   Xu, Zuoshang ^a   Gao, Guangping ^a   Sena Esteves, Miguel ^a  

^a UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

BETA GALACTOSIDASE; GANGLIOSIDE GM1; KETAMINE; XYLAZINE; GANGLIOSIDE; GLB1 PROTEIN, HUMAN;

ADENO ASSOCIATED VIRUS 9; ADULT; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTOSIS; BEHAVIOR; BIOCHEMICAL ANALYSIS; BODY WEIGHT LOSS; BRAIN; BRAIN CORTEX; BRAIN STEM; CENTRAL NERVOUS SYSTEM; CEREBELLUM; CONTROLLED STUDY; DNA EXTRACTION; ENZYMATIC ASSAY; ENZYME ACTIVITY; EXPERIMENTAL BEHAVIORAL TEST; FEMALE; GENE; GENE DOSAGE; GENE EXPRESSION; GENE MUTATION; GENE TRANSFER; GENOTYPE; GLB1 GENE; GM1 GANGLIOSIDOSIS; HEART; HIGH PERFORMANCE LIQUID CHROMATOGRAPHY; HIPPOCAMPUS; HISTOLOGY; INVERTED SCREEN TEST; LIFESPAN; LIMB MOVEMENT; LIQUID CHROMATOGRAPHY-MASS SPECTROMETRY; LIVER; LYSOSOME STORAGE DISEASE; MALE; MASS SPECTROMETRY; MOTOR PERFORMANCE; MOUSE; MUSCLE; NONHUMAN; PARALYSIS; PHENOTYPE; REARING; ROTAROD TEST; SPINAL CORD; SURVIVAL; ULTRA PERFORMANCE LIQUID CHROMATOGRAPHY; ANIMAL; ASTROCYTE; BIOSYNTHESIS; BLOOD; DEPENDOPARVOVIRUS; DISEASE MODEL; GANGLIOSIDOSIS, GM1; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; METABOLISM; PATHOLOGY;

ANIMALS; ASTROCYTES; BETA-GALACTOSIDASE; BRAIN STEM; CENTRAL NERVOUS SYSTEM; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; GANGLIOSIDES; GANGLIOSIDOSIS, GM1; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MICE; SPINAL CORD;

EID: 85016650308     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddv168     Document Type: Article

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