Title : Allogeneic HSCT transfers wild-type cystinosin to nonhematological epithelial cells in cystinosis: First human report.

Pub. Date : 2018 Nov

PMID : 30030899






1 Functional Relationships(s)
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1 We were able to demonstrate successful transfer of the wild-type cystinosin protein and CTNS mRNA to nonhematological epithelial cells in the recipient, as well as a decrease in the tissue cystine-crystal burden. Cystine cystinosin, lysosomal cystine transporter Homo sapiens