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The New Pharmacological Chaperones PBXs Increase α-Galactosidase A Activity in Fabry Disease Cellular Models
Fabry disease is an X-linked multisystemic disorder caused by the impairment of lysosomal α-Galactosidase A, which leads to the progressive accumulation of glycosphingolipids and to defective lysosomal metabolism. Currently, Fabry disease is treated by enzyme replacement therapy or the orally admini...
Autores principales: | Besada, Pedro, Gallardo-Gómez, María, Pérez-Márquez, Tania, Patiño-Álvarez, Lucía, Pantano, Sergio, Silva-López, Carlos, Terán, Carmen, Arévalo-Gómez, Ana, Ruz-Zafra, Aurora, Fernández-Martín, Julián, Ortolano, Saida |
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Formato: | Online Artículo Texto |
Lenguaje: | English |
Publicado: |
MDPI
2021
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Materias: | |
Acceso en línea: | https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8699827/ https://www.ncbi.nlm.nih.gov/pubmed/34944500 http://dx.doi.org/10.3390/biom11121856 |
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