CTNS mRNA as a potential treatment for nephropathic cystinosis
Tjessa Bondue, Sante Princiero Berlingerio, Florian Siegerist, Elena Sendino Garví, Maximilian Schindler, Hans J. Baelde, Sara Cairoli, Bianca Maria Goffredo, Fanny Oliveira Arcolino, Jürgen W. C. Dieker, Manoe Jacoba Janssen, Nicole Endlich, Roland E. Brock, Rik Gijsbers, Lambertus P. van den Heuvel, Elena N. Levtchenko
This paper demonstrates that synthetic CTNS mRNA therapy can restore cystinosin function and reduce cystine accumulation in kidney cells and zebrafish models of cystinosis.
Scientific Reports · Nov 28, 2023Peer-reviewedGenetics