Research Project

Project Title:	mRNA-lipid nanoparticles (mRNA-LNP) for treating cystinosis
Project Type:	Translational research
Disease group(s):	Metabolic & stone disorders
Project Summary:	mRNA-LNP are a new technology to deliver RNA for therapeutic purposes. We aim to test CTNS mRNA-LNPs for treating cystinosis, a lysosomal storage disease due to the CTNS gene mutations. In this translational project we investigate the potential of CTNS mRNA-LNP to correct cystinosis phenotype in cell and animal models of cystinosis.
Lead principal investigator(s):	Elena Levtchenko, Amsterdam Bert van den Heuvel, Nijmegen
Project Period:	10/2020 - 10/2024
Sponsors:	National funding agency