Phosphatidylserine Converts Immunogenic Recombinant Human Acid Alpha-Glucosidase to a Tolerogenic Form in a Mouse Model of Pompe Disease
نویسندگان
چکیده
منابع مشابه
Report of the first Brazilian infantile Pompe disease patient to be treated with recombinant human acid alpha-glucosidase.
OBJECTIVE To describe the first case of infantile Pompe disease to be treated in Brazil. DESCRIPTION Pompe disease is a glycogen storage disease related to defects in the acid alpha-glucosidase enzyme, leading to an intracellular accumulation of glycogen, mainly in muscles. Two forms are described: infantile and juvenile. Since 2006, treatment with recombinant human acid alpha-glucosidase has...
متن کاملLong-term intravenous treatment of Pompe disease with recombinant human alpha-glucosidase from milk.
OBJECTIVE Recent reports warn that the worldwide cell culture capacity is insufficient to fulfill the increasing demand for human protein drugs. Production in milk of transgenic animals is an attractive alternative. Kilogram quantities of product per year can be obtained at relatively low costs, even in small animals such as rabbits. We tested the long-term safety and efficacy of recombinant hu...
متن کاملRecombinant human acid -glucosidase Major clinical benefits in infantile-onset Pompe disease
Background: Pompe disease is a progressive metabolic neuromuscular disorder resulting from deficiency of lysosomal acid -glucosidase (GAA). Infantile-onset Pompe disease is characterized by cardiomyopathy, respiratory and skeletal muscle weakness, and early death. The safety and efficacy of recombinant human (rh) GAA were evaluated in 18 patients with rapidly progressing infantile-onset Pompe d...
متن کاملThe Pharmacological Chaperone AT2220 Increases Recombinant Human Acid α-Glucosidase Uptake and Glycogen Reduction in a Mouse Model of Pompe Disease
Pompe disease is an inherited lysosomal storage disease that results from a deficiency in the enzyme acid α-glucosidase (GAA), and is characterized by progressive accumulation of lysosomal glycogen primarily in heart and skeletal muscles. Recombinant human GAA (rhGAA) is the only approved enzyme replacement therapy (ERT) available for the treatment of Pompe disease. Although rhGAA has been show...
متن کاملAbnormal mannose-6-phosphate receptor trafficking impairs recombinant alpha-glucosidase uptake in Pompe disease fibroblasts
BACKGROUND Pompe disease (PD) is a metabolic myopathy caused by alpha-glucosidase (GAA) deficiency and characterized by generalized glycogen storage. Heterogeneous GAA gene mutations result in wide phenotypic variability, ranging from the severe classic infantile presentation to the milder intermediate and late-onset forms. Enzyme replacement therapy (ERT) with recombinant human GAA (rhGAA), th...
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ژورنال
عنوان ژورنال: Journal of Pharmaceutical Sciences
سال: 2016
ISSN: 0022-3549
DOI: 10.1016/j.xphs.2016.06.018