Decoding Presymptomatic white matter changes in Huntington Disease (WIN-HD)

Lead Research Organisation: University College London
Department Name: Institute of Neurology

Abstract

Huntington disease (HD) is an inherited neurodegenerative disease caused by a single mutation in the huntingtin gene. Onset occurs in adults, with slow progression and a combination of motor signs and cognitive changes, including dementia, as a result of underlying neurodegeneration of brain tissue. The mutation is an expansion of a CAG sequence within the gene and the length of the expansion has a strong effect on disease onset and course.

Early postmortem and imaging studies in HD have focused on grey matter changes in the subcortical brain structures such as the caudate and putamen. However, there is increasing recognition that white matter changes also play a role in the disease from the very early stages, many years before any clinical signs are evident. The exact nature of these white matter changes are not fully understood. Most imaging techniques used to date do not have the power to determine the specific mechanisms underlying white matter degeneration. For example, loss of white matter integrity may be due to degeneration of the axonal section of the neurone, or a reduction in the amount of myelin surrounding it. Myelin is produced by oligodendrocytes and it is thought that dysfunction within these cells may play an important role in the underlying neuropathology in HD.

We aim to improve our understanding of the role of oligodendrocytes in the white matter degeneration which typifies HD by:
a) examining white matter changes in individuals who carry the HD mutation but are not yet affected by the symptoms using advanced neuroimaging techniques
b) using mouse models of HD to examine disease-related changes in living animals (using imaging) and in postmortem brain tissue
c) examining postmortem human brain tissue to understand the nature of white matter changes in terms of characteristics such as axonal loss, demyelination and numbers of oligodendrocytes
d) determining whether other genes which cause dysfunction in oligodendrocytes have an impact on the disease course in HD mouse models

Technical Summary

Our working hypothesis is that oligodendrocyte alterations underlie and exacerbate neurodegeneration in HD. Using new techniques of imaging, neuropathology, cell biology, and genetics, WIN-HD will investigate prodromal white-matter changes in HD patients (22 available human brain tissues and 30 premanifest carriers and 30 gene negative controls for imaging studies) and in mice model. We will use an HD knock-in rodent model and models expressing oligodendrocytes modifiers.

We will test the hypothesis that white-matter changes exacerbate the pathogenic cascade in Huntington Disease (HD) characterized by motor and cognitive signs. These changes may include (1) perturbed axon-glia interactions, (2) loss of oligodendroglial functions, and (3) demyelination. The proposal originates from surprising findings: (1) white-matter atrophy occurs far earlier than clinical onset in HD; and (2) extensive white-matter changes are present in mouse models; and (3) expression of the HD-causing protein in oligodendrocytes may contribute to disease progression in mice. For this project, we have assembled experts in the human disorder, in HD rodent models, in human and animal brain imaging and in the molecular and cellular biology of myelination. Together, we will examine white-matter changes well before the onset of disease signs and look for parallel changes in human and rodent models. We will use diffusion-weighted spectroscopy combined with diffusion tensor imaging to distinguish between axonal and glial contribution to white matter changes. Using genetic tools in mice, we will determine the effects of altered oligodendrocyte function on disease progression. Our two-year program will allow us to infer whether neuroprotective therapies should focus on molecular targets that primarily function in oligodendrocytes.

Planned Impact

Huntington's disease (HD) patients and families, scientists and clinicians working on HD will all be beneficiaries of this research, as well as biotechnology and pharmaceutical companies with research and development and/or commercial interests in these areas. Improving our understanding of the disease-related degeneration of white matter in HD could be an important step forward in developing disease modifying treatments. A number of pharmaceutical and biotechnology companies have existing interests in Huntington's disease, not least because of its genetic and fully penetrant nature make it highly useful in understanding more common neurodegenerative disorders with which its shares many features, such as Alzheimer's disease and Parkinson's disease. Understanding the underlying cellular mechanisms of pathogenesis and seeking to identify new therapeutic targets can only help those endeavours.
The economic and societal burden of neurodegenerative disease is considerable and is certain to increase. Huntington's disease itself affects entire families, with the loss of health in affected individuals having life-changing implications for them, their families and carers. Children in HD families, who themselves may be carrying the disease-causing genetic mutation, may be particularly affected. Furthering our understanding of the pathogenesis of HD will eventually lead to useful disease-modifying treatments. Significant advances in research bring hope to family members and encourage participation in clinical trials.


To maximise the potential gain from our research findings, we will encourage data sharing prior to publication through academic collaboration. Conferences and meetings will help to foster new academic collaborations in addition to those that already exist or have been newly formed as part of this proposal. We plan to hold collaborative review meetings on a regular basis to monitor project progress and identify common themes in neurodegenerative disease research. Our groups benefit from close links with other researchers in their host departments and institutions, and by encouraging greater synergy between groups working on different disorders we hope to identify common mechanisms and means of targeting inflammation in neurodegeneration.

Publications

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Casula EP (2018) Motor cortex synchronization influences the rhythm of motor performance in premanifest huntington's disease. in Movement disorders : official journal of the Movement Disorder Society

 
Description WIN-HD collaboration with Paris 
Organisation Pitié-Salpêtrière Hospital
Country France 
Sector Hospitals 
PI Contribution Participant recruitment, analysis and scientific input on study results and dissemination
Collaborator Contribution Leadership of the project, participant recruitment, analysis and scientific input on study results and dissemination
Impact No output as yet as we are still in data collection phase
Start Year 2016
 
Description 2018 UCL Neuroscience Symposium, London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Postgraduate students
Results and Impact 2018 UCL Neuroscience Symposium, London, UK (June). Presented talk 'Antisense oligonucleotide therapy for Huntington's disease - results from the first HTT lowering clinical trial'. Generated questions and interest in HD trials
Year(s) Of Engagement Activity 2018
 
Description Cotzias Prize Lecture - LXX Annual Meeting of the Spanish Society of Neurology (SEN 2018), Gran Sevilla Events Center, Seville, Spain 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Talk on to Spanish Neurological Society: 'Huntington's disease: natural history, biomarkers and novel therapeutic horizons'
Year(s) Of Engagement Activity 2018
 
Description Discussion leader at 'Molecules to Humans' session at CAG Triplet Repeat Disorders Gordon Research Conference (GRC) Conference, USA 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk titled: 'Molecules to Humans'. Generated questions from audience members
Year(s) Of Engagement Activity 2017
 
Description Distinguished Lecture Series, University of Leuven, Belgium 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Policymakers/politicians
Results and Impact Distinguished Lecture Series, University of Leuven, Belgium. Talk titled: Meeting the therapeutic challenge of Huntington's disease. Generated questions and further interest in research
Year(s) Of Engagement Activity 2019
 
Description FIL Brain Meeting, UCL, London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Postgraduate students
Results and Impact FIL Brain Meeting, The Wellcome Centre for Human Neuroimaging, UCL London, UK. Presented talk 'meeting therapetic challenge of Huntignton's disease'. Talk generated discussion and lively debate.
Year(s) Of Engagement Activity 2018
 
Description Genetic Therapies in Neurodegeneration: Cohorts, Targets and Approaches Symposium, UCL London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Professional Practitioners
Results and Impact Genetic Therapies in Neurodegeneration: Cohorts, Targets and Approaches Symposium, UCL, London, UK (May). Talk titled' Huntington's disease, results from the IONIS HTT Rx CS1 study - the first HTT lowering clinical trial'. Generated questions and interest in HD trials.
Year(s) Of Engagement Activity 2018
 
Description Great Ormond Street Hospital Neurosciences Meeting, London, UK (October) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Professional Practitioners
Results and Impact Great Ormond Street Hospital Neurosciences Meeting, London, UK. Talk titled: Meeting Therapuetic Challenge of Huntington's Disease. Sparked questions and interest in involvement in clinical trials
Year(s) Of Engagement Activity 2018
 
Description Interview for BBC Radio 4 docmentary 'Sliencing the gene' 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Media (as a channel to the public)
Results and Impact Interview on the ongoing Huntington's disease gene-lowering trials to promote research awareness for general public.
Year(s) Of Engagement Activity 2019
URL https://www.bbc.co.uk/sounds/play/m0009rhs
 
Description Interview with Guardian Newspaper 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Interview with the Guardian newspaper regarding IONIS-HTTRx trial results. The interview served to further disseminate trials results and raise the profile of Prof Tabrizi and the UCL HD Centre. The HD Centre was inundated with requests to participate/assist the trial and other Centre led research studies
Year(s) Of Engagement Activity 2017
 
Description Interview with Mosaic Magazine 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Prof Sarah Tabrizi Interview with Mosaic Magazine re ongoing Huntington's disease trials. Serves to raise profile of research and the UCL Huntington's Disease Centre
Year(s) Of Engagement Activity 2017
URL https://mosaicscience.com/story/how-close-are-we-cure-huntingtons/
 
Description Interview/profile in Lancet Neurology 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Professor Sarah Tabrizi completed a profile interview in the Lancet Neurology. Helps raise the profile of her research and the UCL HD Centre.
Year(s) Of Engagement Activity 2017
URL http://www.thelancet.com/journals/laneur/article/PIIS1474-4422(17)30303-4/abstract
 
Description Keynote Lectures at 6th Gordon Research Seminar and Conference on CAG Triplet Repeat Disorders (GRS 2019), Il Ciocco, Italy (June) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Postgraduate students
Results and Impact Produced talk on ongoing titled 'Huntington's disease - trials and tribulations' and molecular pathogenesis to rational therapeutics. Talk presented by colleagues due to personal reasons. Sparked questions and debate.
Year(s) Of Engagement Activity 2019
 
Description Lancet neurology podcast 
Form Of Engagement Activity A broadcast e.g. TV/radio/film/podcast (other than news/press)
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact An invited interview for the Lancet Neurology podcast to further publicise research results on the identification of genetic variants associated with HD progression. Served to wider disseminate results.
Year(s) Of Engagement Activity 2017
URL http://www.thelancet.com/laneur/audio
 
Description Medicine at the Crick Inaugural Event - Gene and Genome Editing Therapy: A new challenge and opportunity in Clinical Medicine, London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Postgraduate students
Results and Impact Medicine at the Crick Inaugural Event - Gene and Genome Editing Therapy: A new challenge and opportunity in Clinical Medicine, London, UK. Talk titled: 'Huntington's disease: RNA targetting therapy and prospects of gene therapy'. Talk generated questions and widened interest in HD research
Year(s) Of Engagement Activity 2018
 
Description Multiple BBC radio broadcasts following IONIS-HTTRx trial results in 11 December 2017 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Multiple BBC Broadcast (TV & Radio Interviews) discussing release IONIS HTTRx trial results. Interviews for: BBC World, BBC News at 6, BBC News at 10, BBC Radio 4, BBC 5 live. The interview served to further disseminate trials results and raise the profile of Prof Tabrizi and the UCL HD Centre. The HD Centre was inundated with requests to participate/assist the trial and other Centre led research studies
Year(s) Of Engagement Activity 2017
URL http://www.bbc.co.uk/news/health-42308341
 
Description Observer news article on Huntington diseae trials 03/03/2019 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Article on Huntington's disease therapies at current clinical trail. https://www.theguardian.com/science/2019/mar/03/huntingtons-disease-new-drug--families. Increased awareness of HD and generated interest in trial.
Year(s) Of Engagement Activity 2019
URL https://www.theguardian.com/science/2019/mar/03/huntingtons-disease-new-drug--families
 
Description Participated in expert panel discussion at Huntington Study Group Conference, 2017 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented short talk on HTT lowering therapies followed by panel discussion and Q&A. Generated questions and wider interest in gene-lowering studies
Year(s) Of Engagement Activity 2007,2017
 
Description Participated in filmed interview for Neuro Central and the Video Journal of Biomedicine 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Filmed interview regarding ongoing HD gene-lowering trials titled 'Genetic therapies for neurodegenerative diseases'.
Year(s) Of Engagement Activity 2019
URL https://www.neuro-central.com/2019/09/12/genetic-therapies-neurodegenerative-diseases-interview-sara...
 
Description Participation on Roche Advisory Board for HD Therapeutic Development 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Expert decisions on Roche therapeutic development strategies for Huntington's disease which influence trial planning
Year(s) Of Engagement Activity 2017
 
Description Plenary Lecture at 15th Annual Update Conference on Clinical Neurology and Neurophysiology, Tel Aviv, Israel (February) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Plenary Lecture at 15th Annual Update Conference on Clinical Neurology and Neurophysiology, Tel Aviv, Israel. Talk title: Meeting the therapeutic challenge of Huntington's Disease. Form genetic modifiers to the first trial of antisense therapy. Talk presented by Prof Lanwehermeyer due Prof Tabrizi illness. Additional talk presented to patient and family groups. Generate questions and further interest in HD research trials.
Year(s) Of Engagement Activity 2019
 
Description Plenary Lecture at 32nd ECNP Congress of Applied and Translational Neuroscience, Copenhagen, Denmark (September) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Presented talk titled 'Novel therapeutic principles against CNS disorders'. Talk sparked questions and discussions and trial enquiries.
Year(s) Of Engagement Activity 2019
 
Description Plenary Lecture at British Society for Genetic Medicine Annual Conference - UK Clinical Genomics 2018, London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Other audiences
Results and Impact Plenary Lecture at British Society for Genetic Medicine Annual Conference - UK Clinical Genomics 2018, London. Talk entitled: `Meeting the therapeutic challenge of Huntington's disease'. Generated questions and interest in research and trials
Year(s) Of Engagement Activity 2018
 
Description Plenary Lecture at the 10th EHDN Plenary Meeting, Vienna, Austria 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Plenary Lecture at the 10th EHDN Plenary Meeting, Vienna, Austria. Presented results of ongoing HTT lowering trials to scientists, patients, industy. Generated questions and interest in developing/participating the clinical trial
Year(s) Of Engagement Activity 2018
 
Description Plenary Lecture at the ABN 2018 Annual Meeting, Birmingham, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Plenary Lecture at the ABN 2018 Annual Meeting, Birmingham, UK. Talk titled: 'Antisense oligonucleotide therapy for Huntington's disease - results from the first HTT lowering clinical trial'. Generated questions and interest in HD trials
Year(s) Of Engagement Activity 2018
 
Description Plenary Lecture at the Alzheimer's Society Annual Conference, London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Plenary Lecture at the Alzheimer's Society Annual Conference, London, UK. Presented talk entitled: Meeting the therapeutic challenge in Huntington's disease and other forms of dementia'
Year(s) Of Engagement Activity 2018
 
Description Plenary Lecture at the UK-Japan Neuroscience Symposium, Royal Society, London, UK (March) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Plenary Lecture at the UK-Japan Neuroscience Symposium, Royal Society, London, UK; Presented talk titled: 'Meeting the therapeutic challenge in Huntington's disease'
Year(s) Of Engagement Activity 2018
 
Description Present talk at European Huntington's Disease Network Conference 2016, The Hague 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk entitled 'Gene targeting therapies for Huntington's disease'. Generated talk questions and audience discussion
Year(s) Of Engagement Activity 2016
 
Description Presentation at Research & Innovation 2019 (ELRIG 2019) Conference, Cambridge (April) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Industry/Business
Results and Impact Presented talk titled Huntingtin lowering strategies for disease modification in Huntington's disease. Sparked questions and discussion.
Year(s) Of Engagement Activity 2019
 
Description Presentation at The Leonard Wolfson Experimental Neurology Centre (LWENC) Research Programme Symposium, London, UK (June) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Postgraduate students
Results and Impact Presented talk titled 'Genetic therapies for Huntington's disease'. Spark questions and discussion
Year(s) Of Engagement Activity 2019
 
Description Presented 6th Annual DCAT lecture 2017, University of Dundee 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Other audiences
Results and Impact Presenting talk entitled 'Meeting the therapeutic challenge in Huntington's disease' to a range of researchers
Year(s) Of Engagement Activity 2017
 
Description Presented Academy of Medical Sciences Jean Shanks Lecture 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Presented lecture titled 'New therapies for Huntington's disease, is the endgame in sight?'. Talk generated questions and discussion
Year(s) Of Engagement Activity 2019
URL https://acmedsci.ac.uk/more/events/annual-fellows-meeting-and-dinner-2019
 
Description Presented Lecture at CHDI Conference in Palm Springs (Feb 2020) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Presented talk titled 'Defining whether there is a boundary of normality decades before clinical onset in Huntington's Disease: the Young Adult Study'
Year(s) Of Engagement Activity 2020
 
Description Presented Seminar to Vertex Pharmaceuticals, Oxford 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Presented talk titled 'Huntington's Disease: An overview' providing overview of HD in the clinic, developments in the field, and insights into potentially fruitful avenues for future therapies.
Year(s) Of Engagement Activity 2017
 
Description Presented Symposium on Neurology, Royal College of Physicians of Edinburgh, Edinburgh, UK (November) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Presented talk titled 'Gene targeting therapies - what does the future hold for neurological disorders?'. Talk sparked discussion and questions
Year(s) Of Engagement Activity 2019
 
Description Presented a talk at the Cambridge Society for the Application of Research 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Postgraduate students
Results and Impact Presented talk titled: 'Gene targeting therapies - what does the future hold for neurological disorders?' Talk generated questions and discussion
Year(s) Of Engagement Activity 2020
 
Description Presented lecture at Birmingham Academic Neurology Seminar, Brimingham (July) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Presented talk titled 'Pioneering genetic therapies for Huntington's Disease'. Talk sparked discussion and debates.
Year(s) Of Engagement Activity 2019
 
Description Presented plenary lecture at CHDI 13th Annual Huntington's Disease Therapeutics Conference: A Forum for Drug Discovery & Development, 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented pleanary talk titled 'Development of IONIS-HTTRx: From first principles to the first successful HTT-lowering drug trial'. Generated questions and sparked numerous requests for participation as well as talks re future plans.
Year(s) Of Engagement Activity 2017
 
Description Presented plenary talk at the The UK-Japan Neuroscience Symposium at the Royal Society, London 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented plenary lecture titled 'Meeting the therapeutic challenge of Huntington's disease' to a wider range of researchers
Year(s) Of Engagement Activity 2017
 
Description Presented poster also selected for oral presentation XXII Congress of the European Society for Stereotactic and Functional Neurosurgery (Madrid, Spain) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented poster titled 'Short-term intensive neurofeedback training using realtime fMri in pre-operative parkinson's disease patients. Jinendra Ekanayake, Marina Papoutsi, Joerg Magerkurth, Oliver Josephs, Catherine Milbao, Marjan Jahanshahi, Patricia Limousin, Tom Foltynie, Geraint Rees, Ludvic Zrinzo'. Poster generated discussions and invite for oral presentation
Year(s) Of Engagement Activity 2016
 
Description Presented poster at European Huntington's Disease Network 2016 Conference, The Hague 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Preseented poster titled 'ASSESSMENT OF IMMUNE SYSTEM ACTIVATION STATUS DURING THE COURSE OF HUNTINGTON'S DISEASE IN HD MOUSE MODELS. Jeffrey Pido-Lopez, Ralph Andre, Agnesska Benjamin, Sophie A. Franklin, Sarah J. Tabrizi and Gillian P. Bates'
Poster generated questions and discussions about future research plans
Year(s) Of Engagement Activity 2016
 
Description Presented poster at European Huntington's Disease Network 2016 Conference, The Hague 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented poster titled: 'Abnormal bioenergetics in inclusion-containing mutant HTT exon 1 primary human neurons. Alison Wood-Kaczmar, Rhia Ghosh, Janos Kriston-Vizi, Edward Smith, Wayne Chadwick, Iain Hargreaves, Simon Heales, Andrey Abramov, Sarah Cole, Robin Ketteler, Gillian Bates, Ralph Andre, Sarah J Tabrizi.
Poster generated questions from attendees and discussions about future research plans.
Year(s) Of Engagement Activity 2016
 
Description Presented poster at European Huntington's Disease Network 2016 Conference, The Hague 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presentation of a poster titled 'Innate transcriptional dysregulation is associated with proinflammatory pathway activation in Huntington's disease myeloid cells. Ralph Andre, James RC Miller, Kitty K Lo, Davina J Hensman Moss, Ulrike Träger, Timothy C Stone, Peter McErlean, Lesley Jones, Peter Holmans, Paul Lavender, Vincent Plagnol and Sarah J Tabrizi
Generated questions from attendees and discussions about future plans
Year(s) Of Engagement Activity 2016
 
Description Presented poster at European Huntington's Disease Network 2016 Conference, The Hague 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented poster:
Ralph Andre1, James RC Miller1, Kitty K Lo2, Davina J Hensman Moss1, Ulrike Träger1#, Timothy C Stone3, Peter McErlean4, Lesley Jones3, Peter Holmans3, Paul Lavender4, Vincent Plagnol2 and Sarah J Tabrizi1. Innate transcriptional dysregulation is associated with proinflammatory pathway activation in Huntington's disease myeloid cells

Generated questions from attendees and discussions about future research plaans
Year(s) Of Engagement Activity 2016
 
Description Presented poster at Hereditary Disease Foundation Conference 2016, Boston 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented poster entitled 'Abnormal bioenergetics in inclusion containing mutant HTT exon 1 primary human neurons'. Generated questions from attendees.
Year(s) Of Engagement Activity 2016
 
Description Presented talk a Lancet Neurology Conference 2017, London 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk entitled 'Gene silencing approaches for Huntington's disease'. Generated questions and audience discussions.

Prof Tabrizi also organised this meeting
Year(s) Of Engagement Activity 2016
 
Description Presented talk at "20th International Congress of Parkinson's Disease and Movement Disorders, Berlin" 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk entitled 'Complex phenotypes and genotypes - seeing the wood for the trees, presentation titled Huntington's disease and HD-like disorders'. Generated audience discussion and questions
Year(s) Of Engagement Activity 2016
 
Description Presented talk at "Wellcome Researcher Meeting 30-31 March 2017: Physiology, Cell Biology and Neuroscience" 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Other audiences
Results and Impact Presented talk entitled 'TREAT-HD: targeting neurodegeneration in Huntington's disease' to other Wellcome Trust funded researchers
Year(s) Of Engagement Activity 2017
 
Description Presented talk at 11th Warsaw International Medical Congress for Young Scientists, Medical University of Warsaw 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Postgraduate students
Results and Impact Presented talk entitled ''Meeting the therapeutic challenge in Huntington's disease'. Generated questions and discussion with audience
Year(s) Of Engagement Activity 2016
 
Description Presented talk at 28th Cambridge Neuroscience Seminar, Cambridge University 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Undergraduate students
Results and Impact Presented talk entitled 'Meeting the therapeutic challenge in Huntington's disease'
Year(s) Of Engagement Activity 2016
 
Description Presented talk at American Neurological Association Conference, Carlsbad 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk titled 'Antisense Oligonucleotide Therapy for Huntington's disease: A Clinical Trials Perspective'' Generated questions from audience and sparked wider discussion post talk
Year(s) Of Engagement Activity 2017
 
Description Presented talk at Association of British Neurologists Special Interest Group Meeting 2017, London 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Presented talk entitled "Silencing abnormal proteins". Generated discussion and questions from the audience
Year(s) Of Engagement Activity 2017
 
Description Presented talk at British Pharmacological Society's annual meeting, London UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Other audiences
Results and Impact Presented talk entitled 'Therapeutic strategies for the treatment of Huntington's disease (and other polyglutamine expansion diseases)' to a range of pharmacology researchers which generated lots of questions
Year(s) Of Engagement Activity 2017
 
Description Presented talk at CAG Triplet Repeat Disorders Gordon Research Conference (GRC) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk entitled 'Meeting the therapeutic challenge of HD'. Generated questions and queries form Audience members
Year(s) Of Engagement Activity 2017
 
Description Presented talk at CASMI / UCLH BRC / UCL Personalised Medicine Domain Symposium, Wednesday 8th March 2017, Institute of Child Health UCL 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Professional Practitioners
Results and Impact Presented talk entitled 'Challenges of accelerating access'
Year(s) Of Engagement Activity 2017
 
Description Presented talk at Division of Brain Sciences, Imperial College London, Charing Cross Hospital 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Postgraduate students
Results and Impact Presented talk entitled "Meeting the Therapeutic Challenge in Huntington's disease". Talk generated questions and audience participation
Year(s) Of Engagement Activity 2016
 
Description Presented talk at European Huntington Association Conference, Sofia 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk titled 'Huntingtin lowering therapies - an update'. Generated questions from audience and requests for more information on trials
Year(s) Of Engagement Activity 2017
 
Description Presented talk at Global Summit on Alzheimer's Disease and other Neurodegenerative Disorders, Lisbon 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Presented talk titled 'Meeting the Therapeutic challenge of Huntington's disease'. Generated questions from the audience widening interest in research
Year(s) Of Engagement Activity 2017
 
Description Presented talk at Hereditary Disease Foundation Annual Gala, 2017, New York, USA 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk titled 'Developing novel therapies for Huntington's disease' towards a largely lay audience.
Year(s) Of Engagement Activity 2017
 
Description Presented talk at Hereditary Disease Foundation, The Milton Wexler Celebration of Life Symposium 2017, Massachusetts 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk entitled 'Early Clinical Development of IONIS-HTTR'. Talk generated questions and audience discussion
Year(s) Of Engagement Activity 2016
 
Description Presented talk at Huntington Study Group Conference and Satellite Symposium, Nashville 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk entitled "Neuroimaging endpoints for HD studies and Track-HD data". Talk generated questions and audience discussion
Year(s) Of Engagement Activity 2016
 
Description Presented talk at Imperial College Neuroscience Society's Conference 2017 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Other audiences
Results and Impact Presented talk titled 'Meeting the therapeutic challenge in Huntington's disease'. Generated questions and audience discussion
Year(s) Of Engagement Activity 2017
 
Description Presented talk at International Congress of Parkinson's Disease and Movement Disorders, International Parkinson and Movement Disorder Society, Nice, France (September) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Presented talk titled 'Current and future therapies for Huntington's disease'. Talk sparked questions, discussions and enquiries about future research
Year(s) Of Engagement Activity 2019
 
Description Presented talk at Network of European Transplantation & Restoration (NECTAR), Dublin, Ireland 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk entitled 'meeting the therapeutic challenge in Huntington's disease' generating questions from other senior researchers.
Year(s) Of Engagement Activity 2017
 
Description Presented talk at Oxford University Departmental Seminar 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Other audiences
Results and Impact Presented talk entitled 'Meeting the Therapeutic Challenge in Huntington's disease'. Generated questions from audience and discussion
Year(s) Of Engagement Activity 2017
 
Description Presented talk at Repair-HD: Working towards stem cell therapies for Huntington's disease Conference, Cardiff 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented talk entitled 'he Ionis study - taking therapeutics interventions to clinic'. Generated questions from audience
Year(s) Of Engagement Activity 2017
 
Description Presented talk at UCL Clinical Genetics Society Spring Conference 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Presented talk entitled 'An update on Huntington's disease and therapy development' to practitioners and post-grad students
Year(s) Of Engagement Activity 2009,2017
 
Description Presented talk at UCL SLMS 2016 Clinical Academic Trainees Symposium, London 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Professional Practitioners
Results and Impact Presented talk entitled 'Gene silencing approaches for Huntington's disease'. Talk generated questions and audience discussions.
Year(s) Of Engagement Activity 2016
 
Description Presented talk at UK HD Network Meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Presented update talk on IONIS-HTTRx trial. Generated questions and request for further information form audience members
Year(s) Of Engagement Activity 2017
 
Description Presented talk at Vertex Pharma Oxford Sciend Day (November) 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Industry/Business
Results and Impact Presented talk: 'Huntington's disease - from bench to bedside'. Talk generated discussion and questions
Year(s) Of Engagement Activity 2019
 
Description Presneted talk at CASMI / UCLH BRC / UCL Personalised Medicine Domain Symposium 2017, London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Public/other audiences
Results and Impact Presented talk entitled 'Cellular and gene therapies'. Generated questions and discussion with audience
Year(s) Of Engagement Activity 2017
 
Description Press conference at American Academy of Neurology Conference, Los Angeles following presentation of HTT lowering phase 1/2a clinical trial results. 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Press conference at American Academy of Neurology Conference following presentation of HTT lowering phase 1/2a clinical trial results. Conference generated questions from media an a plethora of subsequent scientific news articles
Year(s) Of Engagement Activity 2018
 
Description Press release for publication of New England Journal of Medicine paper 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Press release to accompany publication of results from the successful safety trial of a therapeutic for Huntington's disease. The release raised awareness of important research and resulted in enquiries to participate in research.
Year(s) Of Engagement Activity 2019
URL https://www.sciencedaily.com/releases/2019/05/190506150103.htm
 
Description Prof Raymund Roos Farewell Symposium - From bed to bench and back to the future: Huntington's disease, Leiden, The Netherlands 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Prof Raymund Roos Farewell Symposium - From bed to bench and back to the future: Huntington's disease, Leiden, The Netherlands. Presented talk entitled 'The future clinics of Huntington's disease'. Generated questions and sparked debate,
Year(s) Of Engagement Activity 2018
 
Description Royal Society and Academy of Medical Sciences Conference `Delivering Novel Therapies in the 21st Century', London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Royal Society and Academy of Medical Sciences Conference `Delivering Novel Therapies in the 21st Century', London, UK. Presented talk entitled 'ASO Therapies for Huntington's Disease. Results from the first HTT lowering trial'. Talk generated discussion and further interest in clinical trial
Year(s) Of Engagement Activity 2018
 
Description Royal Society of Medicine 17th Medical Innovations Summit, London, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Royal Society of Medicine 17th Medical Innovations Summit, London, UK. Presented talk entiteld 'Meeting the therapeutic challenge of Huntington's disease'. Generated questions and interest in Huntington's disease reserach and trials
Year(s) Of Engagement Activity 2018
 
Description Seminar at UCB Pharma, Belgium 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Talk and roundtable discussion at UCB Pharma on their potential involvement in Huntington's disease research and potential future collaboration.
Year(s) Of Engagement Activity 2018
 
Description UCL Medical Society, London 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Professional Practitioners
Results and Impact UCL Medical Society, London, UK. Presented talk entitled 'meeting therapeutic challenge of Huntington's disease'. Sparked lively debate and widen knowldege and interest in clinical trial
Year(s) Of Engagement Activity 2018
 
Description Wellcome Researcher Meeting: Neuroscience and Mental Health, Coventry, UK 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Wellcome Researcher Meeting: Neuroscience and Mental Health, Coventry, UK. Talk titled: 'Antisense oligonucleotide therapy for Huntington's disease - results from the first HTT lowering clinical trial'.. Generated questions re HD research trials
Year(s) Of Engagement Activity 2018
 
Description Women in Science Seminar Series, University of Sussex Falmer, Brighton, 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Women in Science Seminar Series, University of Science, Falmer, Brighton, UK. Presented talk titled 'meeting therapeutic challenge of Huntignton's disease'. Talk generated questions and interest in HD research
Year(s) Of Engagement Activity 2018
 
Description • DZNE Lecture Series 2018, Bonn, Germany 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Postgraduate students
Results and Impact DZNE Lecture Series 2018, Bonn, Germany (June). Presented talk titled: Meeting the therapeutic challenge in Huntington's disease'. Generated interest in HD trials and questions about further collaboration
Year(s) Of Engagement Activity 2018