In this episode, Dr Sarah Mazelinska speaks with Professor Chris Shaw about his work on the genetics of motor neurone disease and how those discoveries are now being turned into treatments.
They discuss how early clinical experiences shaped his research, the discovery of key genes, and what those findings reveal about how neurodegenerative diseases develop.
The conversation focuses on gene therapy, including the idea of delivering a missing gene directly into the brain using a modified virus. The aim is a one time treatment that could replace long term medication.
They also explore the challenges, including safe delivery, reaching the right cells, and how to control these therapies once given.
The episode closes with a look at where the field is heading, including smarter treatments, combination approaches, and the growing possibility of slowing or stopping disease progression.
Key Points
- MND and frontotemporal dementia share genetic and biological pathways
- Gene therapy can deliver treatment directly into the brain
- The thalamus may act as a central distribution point for therapies
- One time treatments could provide long lasting effects
- Future approaches may combine gene therapy with other treatments
About the Guest
Professor Chris Shaw is a neurologist and researcher at the UK Dementia Research Institute, specialising in the genetics of motor neurone disease and the development of gene therapies.
Dementia Researcher
