Evaluation of the efficacy of cystinosin supplementation through CTNS mRNA delivery in experimental models for cystinosis.
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Bondue T, et al. Among authors: brock r.
Sci Rep. 2023 Nov 28;13(1):20961. doi: 10.1038/s41598-023-47085-w.
Sci Rep. 2023.
PMID: 38016974
Free PMC article.