Moss-Derived Human Recombinant GAA Provides an Optimized Enzyme Uptake in Differentiated Human Muscle Cells of Pompe Disease
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Title
Moss-Derived Human Recombinant GAA Provides an Optimized Enzyme Uptake in Differentiated Human Muscle Cells of Pompe Disease
Authors
Keywords
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Journal
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 21, Issue 7, Pages 2642
Publisher
MDPI AG
Online
2020-04-13
DOI
10.3390/ijms21072642
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- Efficient therapy for refractory Pompe disease by mannose 6-phosphate analogue grafting on acid α-glucosidase
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- Long-term follow-up of 17 patients with childhood Pompe disease treated with enzyme replacement therapy
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- Production and characterization of recombinant human acid α-glucosidase in transgenic rice cell suspension culture
- (2016) Jae-Wan Jung et al. JOURNAL OF BIOTECHNOLOGY
- Survival and long-term outcomes in late-onset Pompe disease following alglucosidase alfa treatment: a systematic review and meta-analysis
- (2016) Benedikt Schoser et al. JOURNAL OF NEUROLOGY
- Mannose receptor-mediated delivery of moss-made α-galactosidase A efficiently corrects enzyme deficiency in Fabry mice
- (2015) Jin-Song Shen et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Characterization of a chemically modified plant cell culture expressed human α-Galactosidase-A enzyme for treatment of Fabry disease
- (2015) Tali Kizhner et al. MOLECULAR GENETICS AND METABOLISM
- Production of a Functional Human Acid Maltase in Tobacco Seeds: Biochemical Analysis, Uptake by Human GSDII Cells, and In Vivo Studies in GAA Knockout Mice
- (2013) Frank Martiniuk et al. APPLIED BIOCHEMISTRY AND BIOTECHNOLOGY
- Glycosylation-independent Lysosomal Targeting of Acid α-Glucosidase Enhances Muscle Glycogen Clearance in Pompe Mice
- (2012) John A. Maga et al. JOURNAL OF BIOLOGICAL CHEMISTRY
- A shortcut to the lysosome: The mannose-6-phosphate-independent pathway
- (2012) Maria Francisca Coutinho et al. MOLECULAR GENETICS AND METABOLISM
- Biotherapeutic target or sink: analysis of the macrophage mannose receptor tissue distribution in murine models of lysosomal storage diseases
- (2011) Xin Sheen Zhang et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Glycoengineered Acid α-Glucosidase With Improved Efficacy at Correcting the Metabolic Aberrations and Motor Function Deficits in a Mouse Model of Pompe Disease
- (2009) Yunxiang Zhu et al. MOLECULAR THERAPY
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