Intravitreal gene therapy reduces lysosomal storage in specific areas of the CNS in mucopolysaccharidosis VII mice

Journal of Neuroscience

Volumn 23, Issue 8, 2003, Pages 3302-3307

  Hennig, Anne K ^a   Levy, Beth ^b   Ogilvie, Judith Mosinger ^a,c   Vogler, Carole A ^b   Galvin, Nancy ^b   Bassnett, Steven ^a   Sands, Mark S ^a  

^a WASHINGTON UNIVERSITY SCHOOL OF MEDICINE   (United States)

^b SAINT LOUIS UNIVERSITY SCHOOL OF MEDICINE   (United States)

^c CENTRAL INSTITUTE FOR THE DEAF   (United States)

Author keywords

Axonal transport; CNS; Gene therapy; Genetic diseases; Inborn errors of metabolism; Lysosomal storage reduction

Indexed keywords

BETA GLUCURONIDASE; CHONDROITIN SULFATE; DERMATAN SULFATE; GLYCOSAMINOGLYCAN POLYSULFATE; HEPARAN SULFATE;

ADENO ASSOCIATED VIRUS; ANIMAL EXPERIMENT; ANIMAL MODEL; ARTICLE; CENTRAL NERVOUS SYSTEM; COGNITIVE DEFECT; ENZYME ACTIVITY; ENZYME DEFICIENCY; GENE THERAPY; HIPPOCAMPUS; LYSOSOME STORAGE DISEASE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; OPTIC TECTUM; PRIORITY JOURNAL; THALAMUS; VIRUS RECOMBINANT; VISUAL CORTEX; VISUAL DISORDER; VITREOUS BODY;

EID: 0037658991     PISSN: 02706474     EISSN: None     Source Type: Journal    
DOI: 10.1523/jneurosci.23-08-03302.2003     Document Type: Article

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