GeneticsScientific ReportsPeer-reviewed
CTNS mRNA as a potential treatment for nephropathic cystinosis
Tjessa Bondue, Sante Princiero Berlingerio, Florian Siegerist…·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.