Effective gene therapy for an inherited CNS disease in a large animal model

Annals of Neurology

Volumn 57, Issue 3, 2005, Pages 355-364

  Vite, Charles H ^a,b   McGowan, Joseph C ^c   Niogi, Sumit ^a,d   Passini, Marco A ^a,b,e   Drobatz, Kenneth J ^a   Haskins, Mark E ^a   Wolfe, John H ^a,b  

^a UNIVERSITY OF PENNSYLVANIA   (United States)

^b CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

^c UNITED STATES NAVAL ACADEMY   (United States)

^d WEILL CORNELL MEDICAL COLLEGE   (United States)

^e GENZYME CORPORATION   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENOVIRUS VECTOR; ALPHA MANNOSIDASE; COMPLEMENTARY DNA; LYSOSOME ENZYME; MESSENGER RNA;

ANIMAL MODEL; ARTICLE; BRAIN TISSUE; CAT; CENTRAL NERVOUS SYSTEM DISEASE; CONTROLLED STUDY; DISEASE SEVERITY; ENZYME ACTIVITY; ENZYME DEFICIENCY; GENE THERAPY; GENETIC DISORDER; IN SITU HYBRIDIZATION; LYSOSOME STORAGE DISEASE; MANNOSIDOSIS; NONHUMAN; NUCLEAR MAGNETIC RESONANCE IMAGING; PRIORITY JOURNAL; TREATMENT OUTCOME; VIRAL GENE THERAPY; VIRUS GENOME;

ALPHA-MANNOSIDASE; ANIMALS; ANIMALS, GENETICALLY MODIFIED; BRAIN; BRAIN MAPPING; CATS; DISEASE MODELS, ANIMAL; GENE THERAPY; GENETIC VECTORS; GLYCOGEN STORAGE DISEASE TYPE II; IN SITU HYBRIDIZATION; INJECTIONS; MAGNETIC RESONANCE IMAGING; NEUROLOGIC EXAMINATION; RNA, MESSENGER; STAINING AND LABELING; TIME FACTORS; TRANSDUCTION, GENETIC; TREATMENT OUTCOME;

EID: 14844284635     PISSN: 03645134     EISSN: None     Source Type: Journal    
DOI: 10.1002/ana.20392     Document Type: Article

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