Biomarkers for disease progression and AAV therapeutic efficacy in feline Sandhoff disease

Experimental Neurology

Volumn 263, Issue , 2015, Pages 102-112

  Bradbury, Allison M ^a   Gray Edwards, Heather L ^a   Shirley, Jamie L ^a   McCurdy, Victoria J ^a   Colaco, Alexandria N ^b   Randle, Ashley N ^a   Christopherson, Pete W ^a   Bird, Allison C ^a   Johnson, Aime K ^a   Wilson, Diane U ^a   Hudson, Judith A ^a   De Pompa, Nicholas L ^a   Sorjonen, Donald C ^a   Brunson, Brandon L ^a   Jeyakumar, Mylvaganam ^b   Platt, Frances M ^b   Baker, Henry J ^a   Cox, Nancy R ^a   Sena Esteves, Miguel ^c   Martin, Douglas R ^a  

^a AUBURN UNIVERSITY   (United States)

^b UNIVERSITY OF OXFORD   (United Kingdom)

^c UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

Author keywords

Biomarkers; Gene therapy; GM2 ganglioside; Hexosaminidase; Lysosomal storage disorder; Neurodegenerative disease

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE; BIOLOGICAL MARKER; GANGLIOSIDE; PARVOVIRUS VECTOR;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLOOD EXAMINATION; CELL COMPARTMENTALIZATION; CELL EXPANSION; CEREBELLUM NUCLEUS; CEREBROSPINAL FLUID ANALYSIS; CONTROLLED STUDY; DISEASE COURSE; DRUG EFFICACY; ELECTRODIAGNOSIS; ENZYME ACTIVITY; GENE; GENE THERAPY; HEX GENE; LATERAL BRAIN VENTRICLE; LYSOSOME; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PERIPHERAL BLOOD MONONUCLEAR CELL; SANDHOFF DISEASE; THALAMUS; ANIMAL; BLOOD; BRAIN; CAT; CEREBROSPINAL FLUID; DEPENDOPARVOVIRUS; DISEASE MODEL; GENE VECTOR; GENETICS; MONONUCLEAR CELL; PATHOLOGY; PROCEDURES;

ANIMALS; BETA-N-ACETYLHEXOSAMINIDASES; BIOMARKERS; BRAIN; CATS; DEPENDOVIRUS; DISEASE MODELS, ANIMAL; DISEASE PROGRESSION; GENETIC THERAPY; GENETIC VECTORS; LEUKOCYTES, MONONUCLEAR; LYSOSOMES; MAGNETIC RESONANCE IMAGING; SANDHOFF DISEASE;

EID: 84908676278     PISSN: 00144886     EISSN: 10902430     Source Type: Journal    
DOI: 10.1016/j.expneurol.2014.09.020     Document Type: Article

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