Project reference: ACE053/22/000086
Total budget: 307 070.04€
Financed by: ACCIO
Summary:
Parkinson’s disease (PD) is a neurodegenerative disorder that affects millions of people worldwide. Understanding its underlying mechanisms and developing effective treatments have been a primary focus of scientific research. In recent years, a significant paradigm shift has occurred in how we approach PD research. This transformation is primarily motivated by ethical and regulatory concerns of the use of animal models, as well as a growing recognition of the limitations of animal models in accurately translating findings to human treatments, especially in the context of drug screening. One promising approach is the use of human cellular models derived from induced pluripotent stem cells (iPSCs).
On one hand, through a genetically modified human iPSC-based cellular system differentiated into dopaminergic neurons, it will be possible to intricately evaluate key molecular and cellular mechanisms in the development of the disease with high translational efficiency and yield. This cellular system will enable large-scale molecule screening assays.
On the other hand, the integration of microfluidics into 3D organoid systems for Parkinson’s disease research represents a promising leap forward. This innovative approach offers a more physiologically relevant, controllable, and efficient platform for studying the intricacies of PD, ultimately bringing us closer to effective treatments and, potentially, a treatment for this debilitating disease.
ZeClinics, led by Dr. Rabadan, has partnered with the CEMIC research group at the University of Barcelona, renowned experts in developing microphysiological platforms using cutting-edge microfluidic technology. The collaborative objective of this proposal is to revolutionize the generation of neural organoids, using human iPSC-derived neurons as the foundational component, while simultaneously constructing a microfluidic chip designed to regulate organoid size and network configuration.
The primary goal is to birth a transformative solution, anticipated to serve as an exceptional research instrument for modeling neuropsychiatric diseases, characterized by a high degree of human relevance. To realize this vision, we will take advantage of patient-derived iPSCs and CRISPR-edited human iPSCs to create a PD neuronal disease model. This innovative approach will combine a novel three-dimensional microfluidic system and sophisticated cell culture developed by cutting-edge biomedical engineering. This system will not only facilitate the study of intricate neuronal processes within Parkinson Disease models but also expedite the testing of therapeutic targets and pharmacological compounds. Importantly, these endeavors will be conducted in a non-invasive, animal-free model, promising a more efficient and humane path towards scientific progress.
Partners:
Universidad de Barcelona, grupo SIC-BIO