DC 10

Gene therapies for cystinosis and cystic fibrosis

About this project

The objective of this PhD project is to develop gene based therapeutic approaches for cystinosis in advanced organoid based models. The PhD candidate will use gene editing (CRISPR-Cas), to build an iPSC derived kidney organoid cystinosis model. Additionally, 3D models like microfluidic plates, hollow fiber membranes, and/or coaxially printed proximal tubules will be used to further model tubulopathy.

The PhD candidate will try to restore gene function in both gut and kidney organoids and developed 3D models. Different assays will be used to evaluate disease genotype for cystinosis and to assess efficacy and safety of the different therapies. We aim to improve gene therapy delivery, which is currently a bottleneck for genetic kidney diseases.

Lead Supervisor: Anne Metje van Genderen 

Host Institution

Lena Heer

Lena Heer

DC10

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