Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy

Journal of Clinical Investigation

Volumn 123, Issue 8, 2013, Pages 3254-3271

  Haurigot, Virginia ^a,b   Marcó, Sara ^a,b   Ribera, Albert ^a,b   Garcia, Miguel ^a,b   Ruzo, Albert ^a,b   Villacampa, Pilar ^a,b   Ayuso, Eduard ^a,b   Añor, Sònia ^a   Andaluz, Anna ^a   Pineda, Mercedes ^c   García Fructuoso, Gemma ^c   Molas, Maria ^a,b   Maggioni, Luca ^a,b   Muñoz, Sergio ^a,b   Motas, Sandra ^a,b   Ruberte, Jess ^a   Mingozzi, Federico ^d   Pumarola, Martí ^a   Bosch, Fatima ^a,b  

^a UNIVERSITAT AUTÒNOMA DE BARCELONA   (Spain)

^b Department of Biochemistry and Molecular Biology ^*  (Spain)

^c HOSPITAL SANT JOAN DE DÉU   (Spain)

^d CHILDREN S HOSPITAL OF PHILADELPHIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ADENO ASSOCIATED VIRUS 9 VECTOR; GLYCOSAMINOGLYCAN; HEPARAN SULFATE; LYSOSOME ENZYME; SULFAMIDASE; SULFATASE; UNCLASSIFIED DRUG; VIRUS VECTOR;

ADENO ASSOCIATED VIRUS; ADENO ASSOCIATED VIRUS 9; ANIMAL EXPERIMENT; ANIMAL MODEL; ANTIBODY TITER; ARTICLE; BIOAVAILABILITY; BLOOD BRAIN BARRIER; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DOSE RESPONSE; ENZYME ACTIVITY; ENZYME REPLACEMENT; FEMALE; GENE DOSAGE; GENE TRANSFER; LYSOSOME STORAGE DISEASE; MALE; MUCOPOLYSACCHARIDOSIS; NONHUMAN; PRIORITY JOURNAL; SANFILIPPO SYNDROME TYPE A; SEROTYPE; THERAPY; VIRAL GENE DELIVERY SYSTEM; VIRAL GENE THERAPY;

EID: 84881228888     PISSN: 00219738     EISSN: 15588238     Source Type: Journal    
DOI: 10.1172/JCI66778     Document Type: Article

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