We use patient-derived human stem cells as a model for investigating the causes of motor neuron disease (ALS) and Parkinson’s disease (PD), with the ultimate aim of translating our insights into improved diagnostics, biomarkers, and novel targets for therapies.
We are both clinician scientists and we jointly lead our research group at the Crick. We use human-induced pluripotent stem cells (hiPSCs) to investigate the key cellular and molecular events underlying neurodegeneration. We examine our human stem cells using cutting-edge dynamic imaging, in order to investigate the mechanisms that lead to neurodegeneration.
We are particularly interested in two key mechanisms: protein misfolding and perturbed RNA regulation, and how these mechanisms influence cell dysfunction during disease.
Overall, our aim is to understand how these two key processes (protein misfolding and perturbed RNA regulation) conspire in neurodegenerative disease. The ultimate hope is that through a better understanding of the molecular and cellular origins of diseases like motor neuron disease (ALS) and Parkinson’s disease (PD), we will be able to take a more strategic approach to the development of novel therapies.