Normalization and improvement of CNS deficits in mice with hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase

Molecular Therapy

Volumn 22, Issue 12, 2014, Pages 2028-2037

  El Amouri, Salim S ^a   Dai, Mei ^a   Han, Jing Fen ^a   Brady, Roscoe O ^b   Pan, Dao ^a,c  

^a CINCINNATI CHILDREN'S HOSPITAL MEDICAL CENTER   (United States)

^b NATIONAL INSTITUTE OF NEUROLOGICAL DISORDERS AND STROKE   (United States)

^c UNIVERSITY OF CINCINNATI COLLEGE OF MEDICINE   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

GLYCOSAMINOGLYCAN; LEVO IDURONIDASE; APOLIPOPROTEIN E; RECEPTOR; RECOMBINANT PROTEIN;

ANIMAL CELL; ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BEHAVIOR DISORDER; BLOOD BRAIN BARRIER; BRAIN METABOLISM; CENTRAL NERVOUS SYSTEM DISEASE; CEREBELLUM; CONTROLLED STUDY; DOSE RESPONSE; DRUG DISTRIBUTION; DRUG DOSE COMPARISON; DRUG EFFECT; DRUG EFFICACY; DRUG MEGADOSE; ENZYME THERAPY; EXPLORATORY BEHAVIOR; HEMATOPOIETIC STEM CELL; HISTOPATHOLOGY; HURLER SYNDROME; LIPID STORAGE; LONG TERM CARE; LOW DRUG DOSE; MOUSE; NONHUMAN; STEM CELL GENE THERAPY; SYSTEMIC THERAPY; ANIMAL; BRAIN; CYTOLOGY; DISEASE MODEL; ENZYMOLOGY; GENE THERAPY; GENE VECTOR; GENETICS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HUMAN; METABOLISM; MUCOPOLYSACCHARIDOSIS I; PATHOLOGY; TISSUE DISTRIBUTION;

MUS;

ANIMALS; APOLIPOPROTEINS E; BLOOD-BRAIN BARRIER; BRAIN; DISEASE MODELS, ANIMAL; GENETIC THERAPY; GENETIC VECTORS; HEMATOPOIETIC STEM CELL TRANSPLANTATION; HEMATOPOIETIC STEM CELLS; HUMANS; IDURONIDASE; MICE; MUCOPOLYSACCHARIDOSIS I; RECEPTORS, PEPTIDE; RECOMBINANT PROTEINS; TISSUE DISTRIBUTION;

EID: 84927178220     PISSN: 15250016     EISSN: 15250024     Source Type: Journal    
DOI: 10.1038/mt.2014.152     Document Type: Article

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