Cysteamine–bicalutamide combination therapy corrects proximal tubule phenotype in cystinosis

Nephropathic cystinosis is a severe monogenic kidney disorder caused by mutations in CTNS, encoding the lysosomal transporter cystinosin, resulting in lysosomal cystine accumulation. The sole treatment, cysteamine, slows down the disease progression, but does not correct the established renal proxim...

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Detalles Bibliográficos
Autores principales: Jamalpoor, Amer, van Gelder, Charlotte AGH, Yousef Yengej, Fjodor A, Zaal, Esther A, Berlingerio, Sante P, Veys, Koenraad R, Pou Casellas, Carla, Voskuil, Koen, Essa, Khaled, Ammerlaan, Carola ME, Rega, Laura Rita, van der Welle, Reini EN, Lilien, Marc R, Rookmaaker, Maarten B, Clevers, Hans, Klumperman, Judith, Levtchenko, Elena, Berkers, Celia R, Verhaar, Marianne C, Altelaar, Maarten, Masereeuw, Rosalinde, Janssen, Manoe J
Formato: Online Artículo Texto
Lenguaje:English
Publicado: John Wiley and Sons Inc. 2021
Materias:
Articles
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8261496/
https://www.ncbi.nlm.nih.gov/pubmed/34165243
http://dx.doi.org/10.15252/emmm.202013067

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8261496/
https://www.ncbi.nlm.nih.gov/pubmed/34165243
http://dx.doi.org/10.15252/emmm.202013067

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