De Geer K, Mascianica K, Naess K, Sardh E, Lindstrand A, Bjorck E
BMC Ophthalmol. 2023; 23(1):394.
PMID: 37752499
PMC: 10523780.
DOI: 10.1186/s12886-023-03136-4.
de Oliveira Poswar F, Henriques Nehm J, Kubaski F, Poletto E, Giugliani R
Ther Clin Risk Manag. 2022; 18:1143-1155.
PMID: 36578769
PMC: 9791935.
DOI: 10.2147/TCRM.S351300.
Dubot P, Sabourdy F, Plat G, Jubert C, Cances C, Broue P
Int J Mol Sci. 2019; 20(21).
PMID: 31661765
PMC: 6861985.
DOI: 10.3390/ijms20215345.
Almandil N, Taha M, Gollapalli M, Rahim F, Ibrahim M, Mosaddik A
BMC Chem. 2019; 13(1):14.
PMID: 31384763
PMC: 6661955.
DOI: 10.1186/s13065-019-0522-x.
Kunihiro A, Luis P, Brickey J, Frye J, Chow H, Schneider C
J Nat Prod. 2019; 82(3):500-509.
PMID: 30794412
PMC: 6528680.
DOI: 10.1021/acs.jnatprod.8b00873.
Neuromuscular degeneration and locomotor deficit in a model of mucopolysaccharidosis VII is attenuated by treatment with resveratrol.
Bar S, Prasad M, Datta R
Dis Model Mech. 2018; 11(11).
PMID: 30459155
PMC: 6262814.
DOI: 10.1242/dmm.036954.
Curcumin, but not curcumin-glucuronide, inhibits Smad signaling in TGFβ-dependent bone metastatic breast cancer cells and is enriched in bone compared to other tissues.
Kunihiro A, Brickey J, Frye J, Luis P, Schneider C, Funk J
J Nutr Biochem. 2018; 63:150-156.
PMID: 30393127
PMC: 6296872.
DOI: 10.1016/j.jnutbio.2018.09.021.
Pathogenesis and treatment of spine disease in the mucopolysaccharidoses.
Peck S, Casal M, Malhotra N, Ficicioglu C, Smith L
Mol Genet Metab. 2016; 118(4):232-43.
PMID: 27296532
PMC: 4970936.
DOI: 10.1016/j.ymgme.2016.06.002.
Native Electrophoresis-Coupled Activity Assays Reveal Catalytically-Active Protein Aggregates of Escherichia coli β-Glucuronidase.
Burchett G, Folsom C, Lane K
PLoS One. 2015; 10(6):e0130269.
PMID: 26121040
PMC: 4484804.
DOI: 10.1371/journal.pone.0130269.
Evaluation of butyrate-induced production of a mannose-6-phosphorylated therapeutic enzyme using parallel bioreactors.
Madhavarao C, Agarabi C, Wong L, Muller-Loennies S, Braulke T, Khan M
Biotechnol Appl Biochem. 2013; 61(2):184-92.
PMID: 24033810
PMC: 10723619.
DOI: 10.1002/bab.1151.
Biochemical evidence for superior correction of neuronal storage by chemically modified enzyme in murine mucopolysaccharidosis VII.
Huynh H, Grubb J, Vogler C, Sly W
Proc Natl Acad Sci U S A. 2012; 109(42):17022-7.
PMID: 23027951
PMC: 3479451.
DOI: 10.1073/pnas.1214779109.
Pyrvinium, a potent small molecule Wnt inhibitor, increases engraftment and inhibits lineage commitment of mesenchymal stem cells (MSCs).
Saraswati S, Deskins D, Holt G, Young P
Wound Repair Regen. 2012; 20(2):185-93.
PMID: 22332749
PMC: 3303160.
DOI: 10.1111/j.1524-475X.2012.00767.x.
Induced pluripotent stem cells derived from mouse models of lysosomal storage disorders.
Meng X, Shen J, Kawagoe S, Ohashi T, Brady R, Eto Y
Proc Natl Acad Sci U S A. 2010; 107(17):7886-91.
PMID: 20385825
PMC: 2867869.
DOI: 10.1073/pnas.1002758107.
Mutations and polymorphisms in GUSB gene in mucopolysaccharidosis VII (Sly Syndrome).
Tomatsu S, Montano A, Dung V, Grubb J, Sly W
Hum Mutat. 2009; 30(4):511-9.
PMID: 19224584
PMC: 3048808.
DOI: 10.1002/humu.20828.
Infused Fc-tagged beta-glucuronidase crosses the placenta and produces clearance of storage in utero in mucopolysaccharidosis VII mice.
Grubb J, Vogler C, Tan Y, Shah G, MacRae A, Sly W
Proc Natl Acad Sci U S A. 2008; 105(24):8375-80.
PMID: 18544647
PMC: 2448844.
DOI: 10.1073/pnas.0803715105.
Lentiviral-transduced human mesenchymal stem cells persistently express therapeutic levels of enzyme in a xenotransplantation model of human disease.
Meyerrose T, Roberts M, Ohlemiller K, Vogler C, Wirthlin L, Nolta J
Stem Cells. 2008; 26(7):1713-22.
PMID: 18436861
PMC: 2736639.
DOI: 10.1634/stemcells.2008-0008.
Chemically modified beta-glucuronidase crosses blood-brain barrier and clears neuronal storage in murine mucopolysaccharidosis VII.
Grubb J, Vogler C, Levy B, Galvin N, Tan Y, Sly W
Proc Natl Acad Sci U S A. 2008; 105(7):2616-21.
PMID: 18268347
PMC: 2268185.
DOI: 10.1073/pnas.0712147105.
Prolonged expression of a lysosomal enzyme in mouse liver after Sleeping Beauty transposon-mediated gene delivery: implications for non-viral gene therapy of mucopolysaccharidoses.
Aronovich E, Bell J, Belur L, Gunther R, Koniar B, Erickson D
J Gene Med. 2007; 9(5):403-15.
PMID: 17407189
PMC: 1868578.
DOI: 10.1002/jgm.1028.
Enzyme therapy in mannose receptor-null mucopolysaccharidosis VII mice defines roles for the mannose 6-phosphate and mannose receptors.
Sly W, Vogler C, Grubb J, Levy B, Galvin N, Tan Y
Proc Natl Acad Sci U S A. 2006; 103(41):15172-7.
PMID: 17015822
PMC: 1622795.
DOI: 10.1073/pnas.0607053103.
External quality assurance programme for enzymatic analysis of lysosomal storage diseases: a pilot study.
Ruijter G, Boer M, Weykamp C, de Vries R, van den Berg I, Janssens-Puister J
J Inherit Metab Dis. 2006; 28(6):979-90.
PMID: 16435191
DOI: 10.1007/s10545-005-0201-z.
