AAV-mediated gene delivery in a feline model of sandhoff disease corrects lysosomal storage in the central nervous system

ASN Neuro

Volumn 7, Issue 2, 2015, Pages

  Rockwell, Hannah E ^a   McCurdy, Victoria J ^b,d   Eaton, Samuel C ^a   Wilson, Diane U ^b   Johnson, Aime K ^b   Randle, Ashley N ^b   Bradbury, Allison M ^b,e   Gray Edwards, Heather L ^b   Baker, Henry J ^b   Hudson, Judith A ^b   Cox, Nancy R ^b   Sena Esteves, Miguel ^c   Seyfried, Thomas N ^a   Martin, Douglas R ^b  

^a BOSTON COLLEGE   (United States)

^b AUBURN UNIVERSITY   (United States)

^c UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^d MISSISSIPPI STATE UNIVERSITY   (United States)

^e UNIVERSITY OF PENNSYLVANIA SCHOOL OF VETERINARY MEDICINE   (United States)

Author keywords

Adeno associated virus; Ganglioside; Gene therapy; Sandhoff disease; hexosaminidase

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE; CEREBROSIDE; CHOLESTEROL ESTER; GANGLIOSIDE GM2; LYSOPHOSPHATIDYLCHOLINE; PARVOVIRUS VECTOR; PHOSPHATIDYLCHOLINE; PHOSPHATIDYLETHANOLAMINE; SPHINGOMYELIN; TRIACYLGLYCEROL; GANGLIO-N-TRIAOSYLCERAMIDE; GANGLIOSIDE; HOMEODOMAIN PROTEIN; SULFATIDE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CAT; CEREBELLUM; CONTROLLED STUDY; CORTICAL BONE; DISEASE COURSE; GENE DELIVERY SYSTEM; HIGH PERFORMANCE THIN LAYER CHROMATOGRAPHY; ION EXCHANGE CHROMATOGRAPHY; LIPID ANALYSIS; LYSOSOME STORAGE DISEASE; NONHUMAN; SANDHOFF DISEASE; THALAMUS; ANIMAL; BRAIN; CENTRAL NERVOUS SYSTEM; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; LYSOSOME; METABOLISM; PATHOLOGY; PATHOPHYSIOLOGY; PROCEDURES; PSYCHOLOGY; QUALITY OF LIFE; SEVERITY OF ILLNESS INDEX; SPINAL CORD; TREATMENT OUTCOME;

ANIMALS; BRAIN; CATS; CENTRAL NERVOUS SYSTEM; CEREBROSIDES; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; G(M2) GANGLIOSIDE; GANGLIOSIDES; GENETIC THERAPY; GENETIC VECTORS; HOMEODOMAIN PROTEINS; LYSOSOMES; QUALITY OF LIFE; SANDHOFF DISEASE; SEVERITY OF ILLNESS INDEX; SPINAL CORD; SULFOGLYCOSPHINGOLIPIDS; TREATMENT OUTCOME;

EID: 84977515780     PISSN: None     EISSN: 17590914     Source Type: Journal    
DOI: 10.1177/1759091415569908     Document Type: Article

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