Stem cell microvesicles transfer cystinosin to human cystinotic cells and reduce cystine accumulation in vitro.

Cystinosis is a rare disease caused by homozygous mutations of the CTNS gene, encoding a cystine efflux channel in the lysosomal membrane. In Ctns knockout mice, the pathologic intralysosomal accumulation of cystine that drives progressive organ damage can be reversed by infusion of wildtype bone ma...

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Bibliografiske detaljer

Principais autores:	Diana M Iglesias, Reyhan El-Kares, Anna Taranta, Francesco Bellomo, Francesco Emma, Martine Besouw, Elena Levtchenko, Jaan Toelen, Lambertus van den Heuvel, Leelee Chu, Jing Zhao, Yoon Kow Young, Nicoletta Eliopoulos, Paul Goodyer
Format:	Artigo
Sprog:	Inglês
Udgivet:	Public Library of Science (PLoS) 2012-01-01
Serier:	PLoS ONE
Online adgang:	https://www.ncbi.nlm.nih.gov/pmc/articles/pmid/22912749/pdf/?tool=EBI
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