Engineering a Paget’s disease stem cell bank
Dr Daniel Scott is leading this research at the University of Nottingham, to engineer a Paget's disease stem cell bank. [2023-2024]
The generated library of cell lines will act as a resource and be held in a collection for the broader Paget’s research community, to incorporate into advanced patient relevant cell models of Paget’s disease, representing a platform to understand and ultimately better target the condition
Paget’s stem cell bank
The recent advent of techniques to generate human induced pluripotent stem cells (iPSCs) – derived from patients – and to differentiate them into theoretically any cell type of the human body has presented a novel means to probe human disease-in‑a‑dish. Concurrently, advances in gene‑editing approaches, for example using CRISPR editing, means that researchers can precisely manipulate patient-derived iPSCs to introduce or ‘correct’ disease-associated gene ‘mutations’ to model how, in isolation, these genetic variants may underlie disease. Collectively, these approaches have revolutionised biomedical research.
With funding from the Paget’s Association and the University of Nottingham, researchers propose to exploit in-house expertise at the Nottingham Paget’s Centre of Excellence, with iPSC culture and CRISPR gene editing, to engineer the first iteration of a Paget’s disease stem cell bank. Specifically, they
will introduce disease-associated mutations into different genes that predispose individuals to Paget’s disease.
The generated library of cell lines will act as a resource and be held in a collection for the broader Paget’s research community, to incorporate into advanced patient relevant cell models of Paget’s disease, representing a platform to understand and ultimately better target the condition.