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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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Detalhes bibliográficos
Publicado no:Pediatr Nephrol
Main Authors: Rocca, Celine J., Cherqui, Stephanie
Formato: Artigo
Idioma:Inglês
Publicado em: 2018
Assuntos:
Acesso em linha: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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