Royal Society - Unravelling the molecular basis of Huntington's disease

Meet world-leading researchers from universities and science institutions across the UK at the Royal Society’s public events of cutting-edge science. Tonight's lecture is "Unravelling the molecular basis of Huntington's disease" as the Royal Society Ferrier Prize Lecture delivered by Professor Gillian Bates FRS.

This livestream event requires prior registration with the Royal Society at the web address below.

https://royalsociety.org/science-events-and-lectures/2025/10/unravelling-the-molecular-basis-of-huntingtons-disease/

SUMMARY
Professor Gillian Bates FRS is awarded the Ferrier Medal 2025 for her work in understanding the molecular basis of Huntington’s disease and consistently producing highly impactful findings which have moulded the course of this field.

This talk covers how in 1993, the mutation that causes Huntington’s disease, a devastating neurodegenerative disorder, was found to be an extra-long CAG repeat in exon 1 of the huntingtin gene (HTT) that encodes a polyglutamine tract in the HTT protein. Over the last 30 years, the Bates lab has been unravelling the first molecular steps by which the mutation causes neuronal dysfunction and neurodegeneration. The mutant CAG repeat is unstable, expanding in specific neuronal cells with age, and the rate of this somatic expansion is known to drive the age of disease onset and rate of disease progression. The Bates lab have found that the mutant HTT pre-mRNA is alternatively processed to generate the small HTT1a transcript, the longer the CAG repeat, the more HTT1a is produced. This encodes the aggregation prone HTT1a protein, that they have shown to be highly pathogenic, representing the second step in the pathogenic cascade. Their recent preclinical studies demonstrate that lowering the HTT1a transcript is much more effective than lowering full-length HTT and may explain the failure of a large huntingtin-lowering clinical trial. Their data indicates that huntingtin-lowering strategies should be designed to target the HTT1a transcript.

SPEAKER
Gillian Bates FRS is Professor of Molecular Neuroscience at the Queen Square Institute of Neurology, UCL and co-directs the Huntington’s Disease Centre with Professor Sarah Tabrizi. She received a first class BSc in Genetics from Sheffield University, an MSc in Biomolecular Organisation from Birkbeck College, and a PhD in the Molecular Genetics of Cystic Fibrosis from Imperial College.

She began her work on Huntington’s Disease (HD) as a postdoctoral scientist when she joined Hans Lehrach’s lab at the ICRF and became a member of the international Huntington’s Disease Collaborative Research Group that identified the HD mutation in 1993. She established her independent lab at KCL and generated the first mouse model of HD in 1996, since which her research has been directed toward understanding the molecular basis of HD and validating therapeutic targets. Since her discovery of the alternatively processed HTT1a transcript in 2013, a major focus of the group has been to investigate how best to design huntingtin-lowering strategies for therapeutic benefit. She was elected to the Academy of Medical Sciences (1999), EMBO (2002) and the Royal Society (2007).

## Attending the event

• The event is free to join, please register via Eventbrite for a ticket
• Live subtitles will be available in-person and virtually

### Attending online

• The lecture will also be livestreamed here and on the Royal Society YouTube channel
• You can take part in the live Q&A via Slido
• This event will be recorded (including the live Q&A) and the recording will be available on YouTube soon after the event

For all enquiries, please contact [awards@royalsociety.org](mailto:awards@royalsociety.org).