https://diler.tube/videos/watch/4dbd0224-6d8f-4d3b-9030-cb9eb8134912 Join us for the Royal Society Ferrier Prize Lecture delivered by Professor Gillian Bates FRS. 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. Sat, 15 Aug 2026 07:13:19 GMT https://validator.w3.org/feed/docs/rss2.html PeerTube - https://diler.tube https://diler.tube/lazy-static/avatars/f58b270a-6076-478a-ad43-3888a532ecb4.png https://diler.tube/videos/watch/4dbd0224-6d8f-4d3b-9030-cb9eb8134912 All rights reserved, unless otherwise specified in the terms specified at https://diler.tube/about and potential licenses granted by each content's rightholder.