Article
Cysteamine-bicalutamide combination treatment restores alpha-ketoglutarate and corrects proximal tubule phenotype in cystinosis
2020-02-11
Abstract excerpt
Nephropathic cystinosis is a severe monogenetic 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 proximal tubulopathy. Here, we developed a new therapeutic strategy by applying an omics-based strategy to expand our knowled...
Topics
Open a Topic to create a Post that cites this publication.
Identifiers and source
- Literature Corpus work
- e74e9b7a-36ab-5192-8650-a3a0130ed593
- DOI
- 10.1101/2020.02.10.941799