Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

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

Volumn 111, Issue 41, 2014, Pages 14870-14875

  Kan, Shih Hsin ^a   Aoyagi Scharber, Mika ^b   Le, Steven Q ^a   Vincelette, Jon ^b   Ohmi, Kazuhiro ^c   Bullens, Sherry ^b   Wendt, Daniel J ^b   Christianson, Terri M ^b   Tiger, Pascale M N ^b   Brown, Jillian R ^b   Lawrence, Roger ^b   Yip, Bryan K ^b   Holtzinger, John ^b   Bagri, Anil ^b   Crippen Harmon, Danielle ^b   Vondrak, Kristen N ^a   Chen, Zhi ^b   Hague, Chuck M ^b   Woloszynek, Josh C ^b   Cheung, Diana S ^b   Webster, Katherine A ^b   Adintori, Evan G ^b   Lo, Melanie J ^b   Wong, Wesley ^b   Fitzpatrick, Paul A ^b   LeBowitz, Jonathan H ^b   Crawford, Brett E ^b   Bunting, Stuart ^b   Dickson, Patricia I ^a   Neufeld, Elizabeth F ^c   more..

^a LOS ANGELES BIOMEDICAL RESEARCH INSTITUTE   (United States)

^b BIOMARIN PHARMACEUTICAL INC   (United States)

^c UNIVERSITY OF CALIFORNIA   (United States)

Author keywords

[No Author keywords available]

Indexed keywords

ACETYLGLUCOSAMINIDASE; ALPHA-N-ACETYL-D-GLUCOSAMINIDASE; BETA N ACETYLHEXOSAMINIDASE; BIOLOGICAL MARKER; HEPARAN SULFATE; HYBRID PROTEIN; LAMP1 PROTEIN, MOUSE; LYSOSOME ASSOCIATED MEMBRANE PROTEIN; PROTEIN BINDING; SOMATOMEDIN B;

ANIMAL; BRAIN; CELL CULTURE; CHO CELL LINE; CRICETULUS; DRUG DELIVERY SYSTEM; ENDOCYTOSIS; FIBROBLAST; HAMSTER; HUMAN; INTRACEREBROVENTRICULAR DRUG ADMINISTRATION; LIVER; METABOLISM; MOUSE; MUCOPOLYSACCHARIDOSIS III; NERVE CELL; PATHOLOGY;

ACETYLGLUCOSAMINIDASE; ANIMALS; BETA-N-ACETYLHEXOSAMINIDASES; BIOMARKERS; BRAIN; CELLS, CULTURED; CHO CELLS; CRICETINAE; CRICETULUS; DRUG DELIVERY SYSTEMS; ENDOCYTOSIS; FIBROBLASTS; HEPARITIN SULFATE; HUMANS; INJECTIONS, INTRAVENTRICULAR; INSULIN-LIKE GROWTH FACTOR II; LIVER; LYSOSOME-ASSOCIATED MEMBRANE GLYCOPROTEINS; MICE; MUCOPOLYSACCHARIDOSIS III; NEURONS; PROTEIN BINDING; RECOMBINANT FUSION PROTEINS;

EID: 84907900752     PISSN: 00278424     EISSN: 10916490     Source Type: Journal    
DOI: 10.1073/pnas.1416660111     Document Type: Article

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