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Potential Use of Stem Cells as a Therapy for Cystinosis

Cystinosis is an autosomal recessive metabolic disease that belongs to the fa1mily of lysosomal storage disorders (LSDs). Initial symptoms of cystinosis correspond to the renal Fanconi syndrome. Patients then develop chronic kidney disease and multi-organ failure due to accumulation of cystine in al...

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Bibliografiske detaljer
Udgivet i:Pediatr Nephrol
Main Authors: Rocca, Celine J., Cherqui, Stephanie
Format: Artigo
Sprog:Inglês
Udgivet: 2018
Fag:
Online adgang:https://ncbi.nlm.nih.gov/pmc/articles/PMC6250595/
https://ncbi.nlm.nih.gov/pubmed/29789935
https://ncbi.nlm.nih.govhttp://dx.doi.org/10.1007/s00467-018-3974-7
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