Recombinant Enzyme Therapy for Fabry Disease: Absence of Editing of Human α-Galactosidase A mRNA

For more than a decade, protein-replacement therapy has been employed successfully for the treatment of Gaucher disease. Recently, a comparable therapy has become available for the related lipid-storage disorder Fabry disease. Two differently produced recombinant α-galactosidase A (α-gal A) preparat...

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Bibliografische gegevens
Gepubliceerd in:Am J Hum Genet
Hoofdauteurs: Blom, Daniël, Speijer, Dave, Linthorst, Gabor E., Donker-Koopman, Wilma G., Strijland, Anneke, Aerts, Johannes M. F. G.
Formaat: Artigo
Taal:Inglês
Gepubliceerd in: Elsevier 2002
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Online toegang:https://ncbi.nlm.nih.govhttps://pmc.ncbi.nlm.nih.gov/articles/PMC420010/
https://ncbi.nlm.nih.govhttps://pubmed.ncbi.nlm.nih.gov/12471562/
https://ncbi.nlm.nih.govhttps://doi.org/10.1086/345309
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