Functional correction of neurological and somatic disorders at later stages of disease in MPS IIIA mice by systemic scAAV9-hSGSH gene delivery

Molecular Therapy Methods and Clinical Development

Volumn 3, Issue , 2016, Pages 16036-

  Fu, Haiyan ^a   Cataldi, Marcela P ^b   Ware, Tierra A ^c   Zaraspe, Kimberly ^a   Meadows, Aaron S ^a   Murrey, Darren A ^a   McCarty, Douglas M ^a,d  

^a THE RESEARCH INSTITUTE AT NATIONWIDE CHILDREN S HOSPITAL   (United States)

^b UNIVERSITY OF NORTH CAROLINA AT CHARLOTTE   (United States)

^c OHIO STATE UNIVERSITY   (United States)

^d THE OHIO STATE UNIVERSITY COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS VECTOR; N SULFOGLUCOSAMINE SULFOHYDROLASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; ASTROCYTOSIS; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DOSE RESPONSE; DRUG DISTRIBUTION; DRUG EFFICACY; ENZYME ACTIVITY; FEMALE; GENE EXPRESSION REGULATION; HUMAN; HUMAN CELL; LIFESPAN; LONGEVITY; MALE; MOUSE; MUCOPOLYSACCHARIDOSIS TYPE 3A; NONHUMAN; PERIPHERAL NERVOUS SYSTEM; PRIORITY JOURNAL; SANFILIPPO SYNDROME; SEROTYPE; SOMATIZATION; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY; GENE DELIVERY SYSTEM; HEK293 CELL LINE; IMMUNOFLUORESCENCE; MORRIS WATER MAZE TEST; MUCOPOLYSACCHARIDOSIS TYPE IIIA; PILOT STUDY; PROTEIN EXPRESSION; TREATMENT OUTCOME;

EID: 84975684050     PISSN: None     EISSN: 23290501     Source Type: Journal    
DOI: 10.1038/mtm.2016.36     Document Type: Article

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