Bondue, Tjessa; Berlingerio, Sante P.; Siegerist, Florian; Sendino-Garví, Elena; Schindler, Maximilian; Cairoli, Sara; Goffredo, Bianca M.; Arcolino, Fanny O.; Dieker, Jürgen; Janssen, Manoe; Endlich, Nicole; Brock, Roland; Gijsbers, Rik; Heuvel, Lambertus van den; Levtchenko, Elena
(bioRxiv, 2023-04-29)
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 correct the genetic defect in nephropathic cystinosis using synthetic ...