Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease.
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R. Mattaliano | Seng H. Cheng | Canwen Jiang | R. Ziegler | T. Edmunds | Jinhua Zhang | W. Canfield | S. Bercury | Yunxiang Zhu | M. Kudo | Karen L Lee | Ji-Lei Jiang | N. Gumlaw
[1] A. Reuser,et al. Pompe's disease , 2008, The Lancet.
[2] R. Sidman,et al. Temporal Neuropathologic and Behavioral Phenotype of 6neo/6neo Pompe Disease Mice , 2008, Journal of neuropathology and experimental neurology.
[3] M. O'Callaghan,et al. Biochemical and pharmacological characterization of different recombinant acid alpha-glucosidase preparations evaluated for the treatment of Pompe disease. , 2008, Molecular genetics and metabolism.
[4] T. Taksir,et al. Ability of adeno-associated virus serotype 8-mediated hepatic expression of acid alpha-glucosidase to correct the biochemical and motor function deficits of presymptomatic and symptomatic Pompe mice. , 2008, Human gene therapy.
[5] P. Doorn,et al. Eight years experience with enzyme replacement therapy in two children and one adult with Pompe disease , 2008, Neuromuscular Disorders.
[6] K. Tsuchida,et al. Transgenic expression of a myostatin inhibitor derived from follistatin increases skeletal muscle mass and ameliorates dystrophic pathology in mdx mice , 2008, FASEB journal : official publication of the Federation of American Societies for Experimental Biology.
[7] C. Vosa,et al. Long-Term Enzyme Replacement Therapy for Pompe Disease With Recombinant Human Alpha-glucosidase Derived From Chinese Hamster Ovary Cells , 2007, Journal of child neurology.
[8] R. Werner,et al. Glycosylation of therapeutic proteins in different production systems , 2007, Acta paediatrica.
[9] R. Desnick,et al. Correction of the Biochemical and Functional Deficits in Fabry Mice Following AAV8-mediated Hepatic Expression of α-galactosidase A. , 2007, Molecular therapy : the journal of the American Society of Gene Therapy.
[10] K. Yarema,et al. Metabolic oligosaccharide engineering: perspectives, applications, and future directions. , 2007, Molecular bioSystems.
[11] M. Ausems,et al. Broad spectrum of Pompe disease in patients with the same c.-32-13T→G haplotype , 2007, Neurology.
[12] B. Byrne,et al. Recombinant human acid α-glucosidase , 2007, Neurology.
[13] E. Ralston,et al. Autophagy and mistargeting of therapeutic enzyme in skeletal muscle in Pompe disease. , 2006, Molecular therapy : the journal of the American Society of Gene Therapy.
[14] B. Thurberg,et al. Characterization of pre- and post-treatment pathology after enzyme replacement therapy for pompe disease , 2006, Laboratory Investigation.
[15] Gary Walsh,et al. Post-translational modifications in the context of therapeutic proteins , 2006, Nature Biotechnology.
[16] G. Herman,et al. Chinese hamster ovary cell-derived recombinant human acid alpha-glucosidase in infantile-onset Pompe disease. , 2006, The Journal of pediatrics.
[17] W. Canfield,et al. Structural Requirements for Efficient Processing and Activation of Recombinant Human UDP-N-acetylglucosamine:Lysosomal-enzyme-N-acetylglucosamine-1-phosphotransferase* , 2006, Journal of Biological Chemistry.
[18] H. Amthor,et al. Molecular mechanisms involving IGF-1 and myostatin to induce muscle hypertrophy as a therapeutic strategy for Duchenne muscular dystrophy. , 2005, Acta myologica : myopathies and cardiomyopathies : official journal of the Mediterranean Society of Myology.
[19] S. Elliott,et al. Glycoengineering: the effect of glycosylation on the properties of therapeutic proteins. , 2005, Journal of pharmaceutical sciences.
[20] N. Raben,et al. Carbohydrate-remodelled acid alpha-glucosidase with higher affinity for the cation-independent mannose 6-phosphate receptor demonstrates improved delivery to muscles of Pompe mice. , 2005, The Biochemical journal.
[21] J. Goldman-Levine,et al. Insulin Detemir—A New Basal Insulin Analog , 2005, The Annals of pharmacotherapy.
[22] K. Nagashima,et al. Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers. , 2005, Molecular therapy : the journal of the American Society of Gene Therapy.
[23] Qun Zhou,et al. Conjugation of Mannose 6-Phosphate-containing Oligosaccharides to Acid α-Glucosidase Improves the Clearance of Glycogen in Pompe Mice* , 2004, Journal of Biological Chemistry.
[24] G. Foster. Pegylated interferons: chemical and clinical differences , 2004, Alimentary pharmacology & therapeutics.
[25] Shigeru Iida,et al. Establishment of FUT8 knockout Chinese hamster ovary cells: An ideal host cell line for producing completely defucosylated antibodies with enhanced antibody‐dependent cellular cytotoxicity , 2004, Biotechnology and bioengineering.
[26] W. Sly,et al. Developmentally regulated mannose 6-phosphate receptor-mediated transport of a lysosomal enzyme across the blood-brain barrier. , 2004, Proceedings of the National Academy of Sciences of the United States of America.
[27] R. Howell,et al. Pompe disease in infants and children. , 2004, The Journal of pediatrics.
[28] W. Kuzon,et al. Satellite cell depletion in degenerative skeletal muscle , 2003, Apoptosis.
[29] R. Brady,et al. Modifying exogenous glucocerebrosidase for effective replacement therapy in Gaucher disease , 1994, Journal of Inherited Metabolic Disease.
[30] S. Iida,et al. Establishment of FUT 8 Knockout Chinese Hamster Ovary Cells : An Ideal Host Cell Line for Producing Completely Defucosylated Antibodies With Enhanced Antibody-Dependent Cellular Cytotoxicity , 2004 .
[31] M. Koury. Sugar coating extends half-lives and improves effectiveness of cytokine hormones. , 2003, Trends in biotechnology.
[32] W. Hop,et al. The natural course of infantile Pompe's disease: 20 original cases compared with 133 cases from the literature. , 2003, Pediatrics.
[33] B. van den Hazel,et al. Glycosylation of an N-terminal extension prolongs the half-life and increases the in vivo activity of follicle stimulating hormone. , 2003, The Journal of clinical endocrinology and metabolism.
[34] Yuqin Wang,et al. Induction of immune tolerance to coagulation factor IX antigen by in vivo hepatic gene transfer. , 2003, The Journal of clinical investigation.
[35] J. Egrie,et al. Darbepoetin alfa has a longer circulating half-life and greater in vivo potency than recombinant human erythropoietin. , 2003, Experimental hematology.
[36] L. Buck,et al. Enhancement of therapeutic protein in vivo activities through glycoengineering , 2003, Nature Biotechnology.
[37] S. Kornfeld,et al. Mannose 6-phosphate receptors: new twists in the tale , 2003, Nature Reviews Molecular Cell Biology.
[38] N. Raben,et al. Glycogen stored in skeletal but not in cardiac muscle in acid alpha-glucosidase mutant (Pompe) mice is highly resistant to transgene-encoded human enzyme. , 2002, Molecular therapy : the journal of the American Society of Gene Therapy.
[39] Qun Zhou,et al. Mannose 6-phosphate quantitation in glycoproteins using high-pH anion-exchange chromatography with pulsed amperometric detection. , 2002, Analytical biochemistry.
[40] P. Trainer,et al. The place of pegvisomant in the management of acromegaly , 2001, Expert opinion on investigational drugs.
[41] R. Kornfeld,et al. Purification and Multimeric Structure of BovineN-Acetylglucosamine-1-phosphodiester α-N-Acetylglucosaminidase* , 1998, The Journal of Biological Chemistry.
[42] H. Reichmann,et al. Adult-onset glycogen storage disease type II: phenotypic and allelic heterogeneity in German patients , 1998, Neurogenetics.
[43] K. Felice,et al. Clinical Variability in Adult‐Onset Acid Maltase Deficiency: Report of Affected Sibs and Review of the Literature , 1995, Medicine.
[44] W. Kiess,et al. Expression of the insulin-like growth factor-II/mannose-6-phosphate receptor in multiple human tissues during fetal life and early infancy. , 1992, The Journal of clinical endocrinology and metabolism.
[45] B. Oostra,et al. Human lysosomal alpha-glucosidase. Characterization of the catalytic site. , 1991, The Journal of biological chemistry.
[46] K. von Figura,et al. Quantitation of Mr 46000 and Mr 300000 mannose 6-phosphate receptors in human cells and tissues. , 1991, Biochemistry international.
[47] D. Livingston,et al. A Modified Human Tissue Plasminogen Activator with Extended Half–Life In Vivo , 1987, Bio/Technology.
[48] B. Brown,et al. Glycogen storage disease type IB. , 1983, Archives of pathology & laboratory medicine.
[49] C. Steer,et al. Uptake and distribution of placental glucocerebrosidase in rat hepatic cells and effects of sequential deglycosylation. , 1981, Biochimica et biophysica acta.