1 |
Tiels P, Baranova E, Piens K et al (2012) A bacterial glycosidase enables mannose-6-phosphate modification and improved cellular uptake of yeast-produced recombinant human lysosomal enzymes. Nat Biotechnol 30, 1225-1231
DOI
|
2 |
Gil JY, Park JN, Lee KJ et al (2015) Increased mannosylphosphorylation of N-glycans by heterologous expression of YlMPO1 in glyco-engineered Saccharomyces cerevisiae for mannose-6-phosphate modification. J Biotechnol 206, 66-74
DOI
|
3 |
Zhu Y, Li X, Kyazike J et al (2004) Conjugation of mannose 6-phosphate-containing oligosaccharides to acid alpha-glucosidase improves the clearance of glycogen in pompe mice. J Biol Chem 279, 50336-50341
DOI
|
4 |
Zhu Y, Li X, McVie-Wylie A et al (2005) Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice. Biochem J 389, 619-628
DOI
|
5 |
Zhu Y, Jiang JL, Gumlaw NK et al (2009) Glycoengineered acid alpha-glucosidase with improved efficacy at correcting the metabolic aberrations and motor function deficits in a mouse model of Pompe disease. Mol Ther 17, 954-963
DOI
|
6 |
Zhou Q, Stefano JE, Harrahy J et al (2011) Strategies for Neoglycan conjugation to human acid alpha-glucosidase. Bioconjug Chem 22, 741-751
DOI
|
7 |
Zhou Q, Avila LZ, Konowicz PA et al (2013) Glycan structure determinants for cation-independent mannose 6-phosphate receptor binding and cellular uptake of a recombinant protein. Bioconjug Chem 24, 2025-2035
DOI
|
8 |
Kishnani PS and Beckemeyer AA (2014) New therapeutic approaches for Pompe disease: enzyme replacement therapy and beyond. Pediatr Endocrinol Rev 12 Suppl 1, 114-124
|
9 |
Chiba Y, Sakuraba H, Kotani M et al (2002) Production in yeast of alpha-galactosidase A, a lysosomal enzyme applicable to enzyme replacement therapy for Fabry disease. Glycobiology 12, 821-828
DOI
|
10 |
Akeboshi H, Kasahara Y, Tsuji D et al (2009) Production of human beta-hexosaminidase A with highly phosphorylated N-glycans by the overexpression of the Ogataea minuta MNN4 gene. Glycobiology 19, 1002-1009
DOI
|
11 |
Tsukimura T, Kawashima I, Togawa T et al (2012) Efficient uptake of recombinant alpha-galactosidase A produced with a gene-manipulated yeast by Fabry mice kidneys. Mol Med 18, 76-82
DOI
|
12 |
Van den Hout JM, Kamphoven JH, Winkel LP et al (2004) Long-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk. Pediatrics 113, e448-457
DOI
|
13 |
Kishnani PS, Nicolino M, Voit T et al (2006) Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease. J Pediatr 149, 89-97
DOI
|
14 |
McVie-Wylie AJ, Lee KL, Qiu H et al (2008) Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease. Mol Genet Metab 94, 448-455
DOI
|
15 |
Tekoah Y, Tzaban S, Kizhner T et al (2013) Glycosylation and functionality of recombinant beta-glucocerebrosidase from various production systems. Biosci Rep 33
DOI
|
16 |
Grabowski GA, Golembo M and Shaaltiel Y (2014) Taliglucerase alfa: an enzyme replacement therapy using plant cell expression technology. Mol Genet Metab 112, 1-8
DOI
|
17 |
Chavez CA, Bohnsack RN, Kudo M, Gotschall RR, Canfield WM and Dahms NM (2007) Domain 5 of the cation-independent mannose 6-phosphate receptor preferentially binds phosphodiesters (mannose 6-phosphate N-acetylglucosamine ester). Biochemistry 46, 12604-12617
DOI
|
18 |
Berger J, Stirnemann J, Bourgne C et al (2012) The uptake of recombinant glucocerebrosidases by blood monocytes from type 1 Gaucher disease patients is variable. Br J Haematol 157, 274-277
DOI
|
19 |
Van Patten SM, Hughes H, Huff MR et al (2007) Effect of mannose chain length on targeting of glucocerebrosidase for enzyme replacement therapy of Gaucher disease. Glycobiology 17, 467-478
DOI
|
20 |
Koeberl DD, Luo X, Sun B et al (2011) Enhanced efficacy of enzyme replacement therapy in Pompe disease through mannose-6-phosphate receptor expression in skeletal muscle. Mol Genet Metab 103, 107-112
DOI
|
21 |
Qian Y, Lee I, Lee WS et al (2010) Functions of the alpha, beta, and gamma subunits of UDP-GlcNAc:lysosomal enzyme N-acetylglucosamine-1-phosphotransferase. J Biol Chem 285, 3360-3370
DOI
|
22 |
Sommerlade HJ, Selmer T, Ingendoh A et al (1994) Glycosylation and phosphorylation of arylsulfatase A. J Biol Chem 269, 20977-20981
|
23 |
Lee K, Jin X, Zhang K et al (2003) A biochemical and pharmacological comparison of enzyme replacement therapies for the glycolipid storage disorder Fabry disease. Glycobiology 13, 305-313
DOI
|
24 |
Sakuraba H, Murata-Ohsawa M, Kawashima I et al (2006) Comparison of the effects of agalsidase alfa and agalsidase beta on cultured human Fabry fibroblasts and Fabry mice. J Hum Genet 51, 180-188
DOI
|
25 |
Lauer RM, Mascarinas T, Racela AS, Diehl AM and Brown BI (1968) Administration of a mixture of fungal glucosidases to a patient with type II glycogenosis (Pompe's disease). Pediatrics 42, 672-676
|
26 |
Sohn Y, Lee JM, Park HR, Jung SC, Park TH and Oh DB (2013) Enhanced sialylation and in vivo efficacy of recombinant human alpha-galactosidase through in vitro glycosylation. BMB Rep 46, 157-162
DOI
|
27 |
Vedder AC, Linthorst GE, Houge G et al (2007) Treatment of Fabry disease: outcome of a comparative trial with agalsidase alfa or beta at a dose of 0.2 mg/kg. PLoS One 2, e598
DOI
|
28 |
Togawa T, Takada M, Aizawa Y, Tsukimura T, Chiba Y and Sakuraba H (2014) Comparative study on mannose 6-phosphate residue contents of recombinant lysosomal enzymes. Mol Genet Metab 111, 369-373
DOI
|
29 |
de Barsy T, Jacquemin P, Van Hoof F and Hers HG (1973) Enzyme replacement in Pompe disease: an attempt with purified human acid alpha-glucosidase. Birth Defects Orig Artic Ser 9, 184-190
|
30 |
Ohashi T (2012) Enzyme replacement therapy for lysosomal storage diseases. Pediatr Endocrinol Rev 10 Suppl 1, 26-34
|
31 |
Ortolano S, Vieitez I, Navarro C and Spuch C (2014) Treatment of lysosomal storage diseases: recent patents and future strategies. Recent Pat Endocr Metab Immune Drug Discov 8, 9-25
DOI
|
32 |
Baldo BA (2015) Enzymes approved for human therapy: indications, mechanisms and adverse effects. BioDrugs 29, 31-55
DOI
|
33 |
Mechler K, Mountford WK, Hoffmann GF and Ries M (2015) Pressure for drug development in lysosomal storage disorders - a quantitative analysis thirty years beyond the US orphan drug act. Orphanet J Rare Dis 10, 46
DOI
|
34 |
Tiede S, Storch S, Lubke T et al (2005) Mucolipidosis II is caused by mutations in GNPTA encoding the alpha/beta GlcNAc-1-phosphotransferase. Nat Med 11, 1109-1112
DOI
|
35 |
Braulke T and Bonifacino JS (2009) Sorting of lysosomal proteins. Biochim Biophys Acta 1793, 605-614
DOI
|
36 |
Kim JJ, Olson LJ and Dahms NM (2009) Carbohydrate recognition by the mannose-6-phosphate receptors. Curr Opin Struct Biol 19, 534-542
DOI
|
37 |
Coutinho MF, Prata MJ and Alves S (2012) Mannose-6- phosphate pathway: a review on its role in lysosomal function and dysfunction. Mol Genet Metab 105, 542-550
DOI
|
38 |
Kudo M, Bao M, D'Souza A et al (2005) The alpha- and beta-subunits of the human UDP-N-acetylglucosamine: lysosomal enzyme N-acetylglucosamine-1-phosphotransferase [corrected] are encoded by a single cDNA. J Biol Chem 280, 36141-36149
DOI
|