Development of a pipeline for the efficacy and safety assessment of gene therapy in microphysiological systems

This project is situated in the development of non-animal, humanized model systems (Organ-on-Chip, OoC). The objectives are: (1) Establish a pipeline for gene therapy delivery screening in OoCs using several first exposure organs (2) Evaluate on target and off target immune reactions covering circulating PBMCs, tissue resident immune cells (system specific) and secondary immune response (lymph node-chip) (3) Evaluate target-cell/tissue specificity and efficacy versus off-target effects and toxicity. The project will involve expertise in the fields of bioengineering, immunology and microfluidics. 

A successful project will result in: (1) Established protocols to evaluate rAAV, VLP and VesiCas in OoC settings. (2) Vector delivery studies including on- and off-target analysis and efficacy using fluorescence-based life imaging on multiple OoC, including: heart, retina, adipose, choroid, pancreas, liver, lymph node and blood vessel. (3) Immune response profiling including circulating immune and tissue resident immune cells.

Enrolment in Doctoral School: University of Tübingen

Planned secondments:

• UCB (M15): analytical methods and QC

• KU Leuven (M15 and M24-25): Vector productions, gene-editing, design of biosensors for life-imaging

• Aristotle University of Thessaloniki (M33-35): Modelling techniques

• Miltenyi Biotech (M38): Working in GMP environments