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Replacement of α-galactosidase A in Fabry disease: effect on fibroblast cultures compared with biopsied tissues of treated patients

The function and intracellular delivery of enzyme therapeutics for Fabry disease were studied in cultured fibroblasts and in the biopsied tissues of two male patients to show diversity of affected cells in response to treatment. In the mutant fibroblasts cultures, the final cellular level of endocyt...

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Main Authors: Keslová-Veselíková, Jana, Hůlková, Helena, Dobrovolný, Robert, Asfaw, Befekadu, Poupětová, Helena, Berná, Linda, Sikora, Jakub, Goláň, Lubor, Ledvinová, Jana, Elleder, Milan
格式: Artigo
語言:Inglês
出版: Springer-Verlag 2008
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在線閱讀:https://ncbi.nlm.nih.gov/pmc/articles/PMC2956889/
https://ncbi.nlm.nih.gov/pubmed/18351385
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00428-008-0586-9
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