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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| Auteurs principaux: | , , , , , , , , , , , , , |
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| Format: | article |
| Langue: | EN |
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Public Library of Science (PLoS)
2012
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| Accès en ligne: | https://doaj.org/article/353a18b5afee4d52adc1fd7c0dda280d |
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