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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Vydáno v:EMBO Mol Med
Hlavní autoři: 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
Médium: Artigo
Jazyk:Inglês
Vydáno: John Wiley and Sons Inc. 2021
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On-line přístup:https://ncbi.nlm.nih.gov/pmc/articles/PMC8261496/
https://ncbi.nlm.nih.gov/pubmed/34165243
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.15252/emmm.202013067
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