ArticleAmerican journal of human genetics2009
Use of a modified alpha-N-acetylgalactosaminidase in the development of enzyme replacement therapy for Fabry disease.
Article in American journal of human genetics, 2009. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 24 papers.
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Who cites it
24 citing papers in PubMed, 53 citations in OpenAlex.
- Therapeutic potential of TMSC-Exo for non-alcoholic fatty liver disease using the liver-on-a-chip model.Journal of translational internal medicine · 2026Article
- Comparative study on incorporation of three recombinant human α-galactosidase A drugs (agalsidases) into cultured fibroblasts and organs/tissues of Fabry mice.Molecular genetics and metabolism reports · 2024Article
- Does administration of hydroxychloroquine/amiodarone affect the efficacy of enzyme replacement therapy for Fabry mice?Molecular genetics and metabolism reports · 2024Article
- Enzymatic properties and clinical associations of serum alpha-galactosidase A in Parkinson's disease.Annals of clinical and translational neurology · 2023Article
- Optimizing human α-galactosidase for treatment of Fabry disease.Scientific reports · 2023Article
- Lysosomal Proteomics Links Disturbances in Lipid Homeostasis and Sphingolipid Metabolism to CLN5 Disease.Cells · 2022Article
- Does administration of hydroxychloroquine/amiodarone accelerate accumulation of globotriaosylceramide and globotriaosylsphingosine in Fabry mice?Molecular genetics and metabolism reports · 2021Article
- Anti-drug antibody formation in Japanese Fabry patients following enzyme replacement therapy.Molecular genetics and metabolism reports · 2020Article
- Plant Glycosides and Glycosidases: A Treasure-Trove for Therapeutics.Frontiers in plant science · 2020Review
- Human Alpha Galactosidases Transiently Produced inFrontiers in plant science · 2017Article
- Fabry Disease: Recognition, Diagnosis, and Treatment of Neurological Features.Current treatment options in neurology · 2016Review
- Protease-resistant modified human β-hexosaminidase B ameliorates symptoms in GM2 gangliosidosis model.The Journal of clinical investigation · 2016Article
- Activities of genes controlling sphingolipid metabolism in human fibroblasts treated with flavonoids.Metabolic brain disease · 2015Article
- Article
- Treatment of lysosomal storage disorders: successes and challenges.Journal of inherited metabolic disease · 2014Review
- Fabry disease: experience of screening dialysis patients for Fabry disease.Clinical and experimental nephrology · 2014Review
- Efficient uptake of recombinant α-galactosidase A produced with a gene-manipulated yeast by Fabry mice kidneys.Molecular medicine (Cambridge, Mass.) · 2012Article
- Mucosal and peripheral Lin- HLA-DR+ CD11c/123- CD13+ CD14- mononuclear cells are preferentially infected during acute simian immunodeficiency virus infection.Journal of virology · 2012Article
- Fabry disease.Orphanet journal of rare diseases · 2010Review
- Interconversion of the specificities of human lysosomal enzymes associated with Fabry and Schindler diseases.The Journal of biological chemistry · 2010Article
Corrections and comments
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Authors and funding
15 authors at 6 institutions in 1 country.
Funding
No grant is acknowledged in the PubMed record.
Abstract
A modified alpha-N-acetylgalactosaminidase (NAGA) with alpha-galactosidase A (GLA)-like substrate specificity was designed on the basis of structural studies and was produced in Chinese hamster ovary cells. The enzyme acquired the ability to catalyze the degradation of 4-methylumbelliferyl-alpha-D-galactopyranoside. It retained the original NAGA's stability in plasma and N-glycans containing many mannose 6-phosphate (M6P) residues, which are advantageous for uptake by cells via M6P receptors. There was no immunological cross-reactivity between the modified NAGA and GLA, and the modified NAGA did not react to serum from a patient with Fabry disease recurrently treated with a recombinant GLA. The enzyme cleaved globotriaosylceramide (Gb3) accumulated in cultured fibroblasts from a patient with Fabry disease. Furthermore, like recombinant GLA proteins presently used for enzyme replacement therapy (ERT) for Fabry disease, the enzyme intravenously injected into Fabry model mice prevented Gb3 storage in the liver, kidneys, and heart and improved the pathological changes in these organs. Because this modified NAGA is hardly expected to cause an allergic reaction in Fabry disease patients, it is highly promising as a new and safe enzyme for ERT for Fabry disease.
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Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.