Biochemical, histological and functional correction of mucopolysaccharidosis Type IIIB by intra-cerebrospinal fluid gene therapy

Human Molecular Genetics

Volumn 24, Issue 7, 2014, Pages 2078-2095

  Ribera, Albert ^a   Haurigot, Virginia ^a   Garcia, Miguel ^a   Marcó, Sara ^a   Motas, Sandra ^a   Villacampa, Pilar ^a   Maggioni, Luca ^a   León, Xavier ^a   Molas, Maria ^a   Sánchez, Víctor ^a   Muñoz, Sergio ^a   Leborgne, Christian ^b   Moll, Xavier ^a   Pumarola, Martí ^a   Mingozzi, Federico ^b,c   Ruberte, Jesús ^a   Añor, Sònia ^a   Bosch, Fatima ^a  

^a UNIVERSITAT AUTÒNOMA DE BARCELONA   (Spain)

^b GÉNÉTHON   (France)

^c SORBONNE UNIVERSITÉ   (France)

Author keywords

[No Author keywords available]

Indexed keywords

ALPHA N ACETYLGLUCOSAMINIDASE; GLYCOSAMINOGLYCAN; PARVOVIRUS VECTOR; ACETYLGLUCOSAMINIDASE; ALPHA-N-ACETYL-D-GLUCOSAMINIDASE;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; CEREBROSPINAL FLUID; CONTROLLED STUDY; ENZYME ACTIVITY; GENE EXPRESSION; GENE THERAPY; GENE TRANSFER; HOMEOSTASIS; LYSOSOME; MALE; MICROGLIA; MOUSE; MUCOPOLYSACCHARIDOSIS; MUCOPOLYSACCHARIDOSIS TYPE 3B; NERVOUS SYSTEM INFLAMMATION; NONHUMAN; PRIORITY JOURNAL; ANIMAL; BRAIN; C57BL MOUSE; DEPENDOPARVOVIRUS; ENZYMOLOGY; EVALUATION STUDY; FEMALE; GENE VECTOR; GENETICS; HUMAN; METABOLISM; MUCOPOLYSACCHARIDOSIS III; PATHOLOGY; PROCEDURES;

ANIMALIA; CANIS FAMILIARIS; MUS;

ACETYLGLUCOSAMINIDASE; ANIMALS; BRAIN; DEPENDOVIRUS; FEMALE; GENETIC THERAPY; GENETIC VECTORS; HUMANS; MALE; MICE; MICE, INBRED C57BL; MUCOPOLYSACCHARIDOSIS III;

EID: 84926453574     PISSN: 09646906     EISSN: 14602083     Source Type: Journal    
DOI: 10.1093/hmg/ddu727     Document Type: Article

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