Article
Cell-Based Phenotypic Drug Screening Identifies Luteolin as Candidate Therapeutic for Nephropathic Cystinosis.
Journal of the American Society of Nephrology : JASN - 1 Jul 2020
De Leo Ester, Elmonem Mohamed A, Berlingerio Sante Princiero, Berquez Marine, Festa Beatrice Paola, Raso Roberto, Bellomo Francesco, Starborg Tobias, Janssen Manoe Jacoba, Abbaszadeh Zeinab, Cairoli Sara, Goffredo Bianca Maria, Masereeuw Rosalinde, Devuyst Olivier, Lowe Martin, Levtchenko Elena, Luciani Alessandro, Emma Francesco, Rega Laura Rita
Abstract excerpt
BACKGROUND: Mutations in the gene that encodes the lysosomal cystine transporter cystinosin cause the lysosomal storage disease cystinosis. Defective cystine transport leads to intralysosomal accumulation and crystallization of cystine. The most severe phenotype, nephropathic cystinosis, manifests during the first months of life, as renal Fanconi syndrome. The cystine-depleting agent cysteamine significantly...
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