Overcoming the blood-brain barrier with high-dose enzyme replacement therapy in murine mucopolysaccharidosis VII

Proceedings of the National Academy of Sciences of the United States of America

Volumn 102, Issue 41, 2005, Pages 14777-14782

  Vogler, Carole ^a   Levy, Beth ^a   Grubb, Jeffrey H ^a   Galvin, Nancy ^a   Tan, Yun ^a   Kakkis, Emil ^b,c   Pavloff, Nadine ^c   Sly, William S ^a  

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

^b HARBOR UCLA MEDICAL CENTER   (United States)

^c BIOMARIN PHARMACEUTICAL INC   (United States)

Author keywords

glucuronidase deficiency; Immune tolerance; Lysosomal storage disease; Mannose 6 phosphate receptor

Indexed keywords

BETA GLUCURONIDASE; RECOMBINANT ENZYME;

ANIMAL EXPERIMENT; ANIMAL MODEL; ANIMAL TISSUE; ARTICLE; BLOOD BRAIN BARRIER; CENTRAL NERVOUS SYSTEM; CONTROLLED STUDY; DRUG MEGADOSE; ENZYME DEFICIENCY; ENZYME REPLACEMENT; IMMUNOLOGICAL TOLERANCE; LYSOSOME STORAGE DISEASE; MOUSE; MUCOPOLYSACCHARIDOSIS; MUCOPOLYSACCHARIDOSIS TYPE 7; NONHUMAN; PRIORITY JOURNAL;

ANIMALS; BLOOD-BRAIN BARRIER; DOSE-RESPONSE RELATIONSHIP, DRUG; GLUCURONIDASE; HISTOLOGICAL TECHNIQUES; HUMANS; MENINGES; MICE; MICE, TRANSGENIC; MUCOPOLYSACCHARIDOSIS VII; NEUROGLIA; NEURONS;

MURINAE;

EID: 26844534412     PISSN: 00278424     EISSN: None     Source Type: Journal    
DOI: 10.1073/pnas.0506892102     Document Type: Article

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