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Cystinosis: Therapeutic implications of in vitro studies of cultured fibroblasts

Authors
Journal
The Journal of Pediatrics
0022-3476
Publisher
Elsevier
Publication Date
Volume
78
Issue
5
Identifiers
DOI: 10.1016/s0022-3476(71)80355-4
Disciplines
  • Medicine

Abstract

Cultured skin fibroblasts from patients with nephropathic cystine storage disease were grown in media with different cystine concentrations. The size of the intracellular cystine pool in these cells and, more strikingly, the rate of cystine influx into this pool were directly correlated with the extracellular cystine level. The results have implications for therapeutic approaches to cystinosis based on lowering the plasma cystine level. A scientific rationale for such therapy is proposed, but the degree of efficacy of treatment cannot be predicted from these in vitro studies.

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