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


National funding agency

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