Mucopolysaccharidosis-like phenotype in feline Sandhoff disease and partial correction after AAV gene therapy

Molecular Genetics and Metabolism

Volumn 116, Issue 1-2, 2015, Pages 80-87

  Gray Edwards, Heather L ^a   Brunson, Brandon L ^a   Holland, Merrilee ^a   Hespel, Adrien Maxence ^a   Bradbury, Allison M ^a,e   McCurdy, Victoria J ^a,f   Beadlescomb, Patricia M ^a   Randle, Ashley N ^a   Salibi, Nouha ^b,c   Denney, Thomas S ^c   Beyers, Ronald J ^c   Johnson, Aime K ^a   Voyles, Meredith L ^a   Montgomery, Ronald D ^a   Wilson, Diane U ^a   Hudson, Judith A ^a   Cox, Nancy R ^a   Baker, Henry J ^a   Sena Esteves, Miguel ^d   Martin, Douglas R ^a  

^a AUBURN UNIVERSITY   (United States)

^b SIEMENS CORPORATE RESEARCH   (United States)

^c AUBURN UNIVERSITY   (United States)

^d UNIVERSITY OF MASSACHUSETTS MEDICAL SCHOOL   (United States)

^e UNIVERSITY OF PENNSYLVANIA   (United States)

^f MISSISSIPPI STATE UNIVERSITY   (United States)

Author keywords

Bone; Gene therapy; GM2 gangliosidosis; Heart; Lysosomal storage disease; Sandhoff disease

Indexed keywords

BETA N ACETYLHEXOSAMINIDASE; COMPLEMENTARY DNA; GLYCOSAMINOGLYCAN; PARVOVIRUS VECTOR;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BRAIN CORTEX; CAT; CATABOLISM; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DISEASE SEVERITY; ECHOCARDIOGRAPHY; FIBROBLAST; HISTOPATHOLOGY; LIVER; MUCOPOLYSACCHARIDOSIS; MUTATIONAL ANALYSIS; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PHENOTYPE; PRIORITY JOURNAL; RADIOGRAPHY; SANDHOFF DISEASE; SPINAL CORD COMPRESSION; SYMPTOMATOLOGY; VIRAL GENE THERAPY; ADENOVIRIDAE; ANIMAL; ANIMAL STRUCTURES; DISEASE MODEL; GENE THERAPY; GENE VECTOR; GENETICS; HUMAN; MUCOPOLYSACCHARIDOSES; PATHOLOGY; PATHOPHYSIOLOGY; URINE;

ADENOVIRIDAE; ANIMAL STRUCTURES; ANIMALS; BETA-N-ACETYLHEXOSAMINIDASES; CATS; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MUCOPOLYSACCHARIDOSES; PHENOTYPE; SANDHOFF DISEASE;

EID: 84940889857     PISSN: 10967192     EISSN: 10967206     Source Type: Journal    
DOI: 10.1016/j.ymgme.2015.05.003     Document Type: Article

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