Developing models to understand and treat MND using induced pluripotent stem cells (iPSCs)

Project summary

Neurones are particularly active cells that continuously send signals around the body; this large workload places a considerable metabolic load on the neurones. If the energy demands of the neurones are not met, they will ultimately falter, and die.

In this project, we are developing iPSC-derived neurones from MND and non-MND subjects to assess neuronal bioenergetics. Insights gained from these iPSC-derived neurones will allow us to determine the impact of neuronal bioenergetics on disease pathogenesis. Using these iPSC-derived neurones, we aim to develop a high-throughput platform that will allow us to screen potential therapeutic candidates in MND, with the view to prevent neuronal death and thus, halt or slow disease progression in MND.

We are also using iPSC-derived neurones and organoid models to decipher the molecular mechanisms that contribute to cell death. This includes the assessment of mitochondrial dynamics, and the use of RNA-sequencing technologies to identify gene signatures that can inform about disease processes.