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Glycosylation-independent targeting enhances enzyme delivery to lysosomes and decreases storage in mucopolysaccharidosis type VII mice.

LeBowitz JH, Grubb JH, Maga JA, Schmiel DH, Vogler C, Sly WS.

Proc Natl Acad Sci U S A. 2004 Mar 2;101(9):3083-8. Epub 2004 Feb 19.


Acidic amino acid tag enhances response to enzyme replacement in mucopolysaccharidosis type VII mice.

Montaño AM, Oikawa H, Tomatsu S, Nishioka T, Vogler C, Gutierrez MA, Oguma T, Tan Y, Grubb JH, Dung VC, Ohashi A, Miyamoto K, Orii T, Yoneda Y, Sly WS.

Mol Genet Metab. 2008 Jun;94(2):178-89. doi: 10.1016/j.ymgme.2008.01.007. Epub 2008 Mar 21.


New strategies for enzyme replacement therapy for lysosomal storage diseases.

Grubb JH, Vogler C, Sly WS.

Rejuvenation Res. 2010 Apr-Jun;13(2-3):229-36. doi: 10.1089/rej.2009.0920. Review.


Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors.

Sly WS, Vogler C, Grubb JH, Levy B, Galvin N, Tan Y, Nishioka T, Tomatsu S.

Proc Natl Acad Sci U S A. 2006 Oct 10;103(41):15172-7. Epub 2006 Oct 2.


Enzyme replacement in murine mucopolysaccharidosis type VII: neuronal and glial response to beta-glucuronidase requires early initiation of enzyme replacement therapy.

Vogler C, Levy B, Galvin NJ, Thorpe C, Sands MS, Barker JE, Baty J, Birkenmeier EH, Sly WS.

Pediatr Res. 1999 Jun;45(6):838-44.


Insulin-like growth factor II peptide fusion enables uptake and lysosomal delivery of α-N-acetylglucosaminidase to mucopolysaccharidosis type IIIB fibroblasts.

Kan SH, Troitskaya LA, Sinow CS, Haitz K, Todd AK, Di Stefano A, Le SQ, Dickson PI, Tippin BL.

Biochem J. 2014 Mar 1;458(2):281-9. doi: 10.1042/BJ20130845.


Murine mucopolysaccharidosis type VII: the impact of therapies on the clinical course and pathology in a murine model of lysosomal storage disease.

Vogler C, Sands MS, Galvin N, Levy B, Thorpe C, Barker J, Sly WS.

J Inherit Metab Dis. 1998 Aug;21(5):575-86. Review.


Infused Fc-tagged beta-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice.

Grubb JH, Vogler C, Tan Y, Shah GN, MacRae AF, Sly WS.

Proc Natl Acad Sci U S A. 2008 Jun 17;105(24):8375-80. doi: 10.1073/pnas.0803715105. Epub 2008 Jun 10.


Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.

Huynh HT, Grubb JH, Vogler C, Sly WS.

Proc Natl Acad Sci U S A. 2012 Oct 16;109(42):17022-7. doi: 10.1073/pnas.1214779109. Epub 2012 Oct 1.


Chemically modified beta-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII.

Grubb JH, Vogler C, Levy B, Galvin N, Tan Y, Sly WS.

Proc Natl Acad Sci U S A. 2008 Feb 19;105(7):2616-21. doi: 10.1073/pnas.0712147105. Epub 2008 Feb 11.


Glycosylation-independent lysosomal targeting of acid α-glucosidase enhances muscle glycogen clearance in pompe mice.

Maga JA, Zhou J, Kambampati R, Peng S, Wang X, Bohnsack RN, Thomm A, Golata S, Tom P, Dahms NM, Byrne BJ, LeBowitz JH.

J Biol Chem. 2013 Jan 18;288(3):1428-38. doi: 10.1074/jbc.M112.438663. Epub 2012 Nov 27.


Numerous transcriptional alterations in liver persist after short-term enzyme-replacement therapy in a murine model of mucopolysaccharidosis type VII.

Woloszynek JC, Roberts M, Coleman T, Vogler C, Sly W, Semenkovich CF, Sands MS.

Biochem J. 2004 Apr 15;379(Pt 2):461-9.


Enzyme replacement with recombinant beta-glucuronidase in the newborn mucopolysaccharidosis type VII mouse.

Vogler C, Sands M, Higgins A, Levy B, Grubb J, Birkenmeier EH, Sly WS.

Pediatr Res. 1993 Dec;34(6):837-40.


Transgene produces massive overexpression of human beta -glucuronidase in mice, lysosomal storage of enzyme, and strain-dependent tumors.

Vogler C, Galvin N, Levy B, Grubb J, Jiang J, Zhou XY, Sly WS.

Proc Natl Acad Sci U S A. 2003 Mar 4;100(5):2669-73. Epub 2003 Feb 18.


Recombinant adeno-associated virus-mediated correction of lysosomal storage within the central nervous system of the adult mucopolysaccharidosis type VII mouse.

Sferra TJ, Qu G, McNeely D, Rennard R, Clark KR, Lo WD, Johnson PR.

Hum Gene Ther. 2000 Mar 1;11(4):507-19.


Defining the pathway for Tat-mediated delivery of beta-glucuronidase in cultured cells and MPS VII mice.

Orii KO, Grubb JH, Vogler C, Levy B, Tan Y, Markova K, Davidson BL, Mao Q, Orii T, Kondo N, Sly WS.

Mol Ther. 2005 Aug;12(2):345-52.

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