Stem cells for human disease modelling and potential for tissue repair

Title : Stem cells for human disease modelling and potential for tissue repair

Abstract:

Understanding the mechanisms underlying human disease and translating this knowledge into therapeutic strategies remain major challenges, particularly for congenital disorders that affect multiple tissues and organs. A key issue is the importance of selecting the most appropriate cellular and experimental model for the biological question being addressed and for developing novel strategies for tissue repair. 

Advances in patient-derived cellular models, including primary cells and induced pluripotent stem cells (iPSCs), have provided new opportunities to investigate disease processes in relevant human cell types and tissues. At the same time, the expanding range of culture systems available, from conventional two-dimensional cultures to three-dimensional self-organising or biomaterial-supported engineered tissue models, raises important questions about how best to investigate different aspects of human diseases and potential therapies.

Drawing on examples from our work on congenital disorders including microtia, acrodysostosis and Duchenne muscular dystrophy, I will explore how the choice of cell source and culture environment influences our ability to elucidate pathogenic mechanisms and engineer tissues. I will discuss what different patient-derived cell-based models can tell us about human disease, their respective strengths and limitations, and the types of questions they are best suited to address.

Finally, I will consider how insights gained from human disease modelling inform the broader goal of repairing diseased tissue and restoring function. While stem cell-based approaches hold considerable promise for future therapies and regenerative medicine, significant biological, technical and translational challenges remain. Understanding both the strengths and limitations of current models will be essential for maximising their potential impact on improving outcome for congenital disorders and other conditions affecting human health.

Biography:

Patrizia Ferretti is Professor of Regenerative Biology at UCL Great Ormond Street Institute of Child Health, University College London, UK. She studied at Pisa University and at the Mario Negri Institute for Pharmacological Research, Milan, Italy, prior to moving to the Max Planck Institute, Goettingen, Germany, and then to London. Her group has made sustained and internationally recognized contributions to the regeneration and rare disease field, by identifying cellular and molecular mechanisms which play a role in response to injury and repair and establishing human disease models using stem/progenitor cells. She has published over 130 papers.