About this project

This PhD project will focus on using messenger RNA (mRNA) as a relatively safe (non-integrating and non-permanent) model therapeutic, that ultimately delivers a novel treatment option for cystinosis and a base-system to build next-generation permanent gene therapies for cystinosis and cystic fibrosis.

The PhD candidate will develop novel mRNAs and validate Mercurna’s in-house developed targeting lipid nanoparticles (LNPs) using the ORGESTRA organoid models.

Next to design and analysis of functional delivery, we also evaluate the safety and efficacy of such mRNA-LNPs.

Lead Supervisor: Jenny van Asbeck-van der Wijst