Evaluation of the efficacy of cystinosin supplementation through CTNS mRNA delivery in experimental models for cystinosis

Messenger RNA (mRNA) therapies are emerging in different disease areas, but have not yet reached the kidney field. Our aim was to study the feasibility to treat the genetic defect in cystinosis using synthetic mRNA in cell models and ctns-/- zebrafish embryos. Cystinosis is a prototype lysosomal sto...

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Veröffentlicht in:	SCIENTIFIC REPORTS 2023-11, Vol.13 (1)
Hauptverfasser:	Bondue, Tjessa, Berlingerio, Sante Princiero, Siegerist, Florian, Sendino-Garvi, Elena, Schindler, Maximilian, Baelde, Hans Jacobus, Cairoli, Sara, Goffredo, Bianca Maria, Arcolino, Fanny Oliveira, Dieker, Jurgen, Janssen, Manoe Jacoba, Endlich, Nicole, Brock, Roland, Gijsbers, Rik, van den Heuvel, Lambertus, Levtchenko, Elena
Format:	Artikel
Sprache:	eng
Online-Zugang:	Volltext
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