Targeting the innate immune system in Huntington's disease

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

Abstract

Huntington's disease (HD) is a fatal, inherited neurological condition. The earliest symptoms are often subtle problems with mood or cognition, followed by a lack of coordination and an unsteady gait. As the disease progresses, jerky body movements become more apparent, along with a decline in mental abilities and behavioural and psychiatric problems. Affected individuals also suffer progressive weight loss and muscle wasting. The disease typically progresses over several decades until death. There are a few effective treatments for some of the symptoms, but there is no cure that will halt or slow progression of the disease. The inherited nature of HD means that the children of affected individuals have a 50% chance of developing the disease themselves.

The disease is caused by a mutation in a single gene that encodes for a protein called huntingtin (HTT). The only difference between the mutated and normal versions of the gene is an increase in the number of repeats of the DNA sequence CAG close to its beginning. This causes an increase in the number of glutamines (an amino acid, the building blocks of proteins) in the HTT protein. This causes the HTT protein to behave in an aberrant fashion in the cells in which it's made.

HD is primarily thought of as disease of the brain, but the HTT protein (both the mutant and normal forms) is expressed in all the cells of the body. It is becoming increasingly recognised that there are significant changes outside of the brain in HD, including in muscle, heart, fat and, of interest here, the immune system. In fact, people carrying the HD gene show signs of increased inflammation, as shown by elevated levels of certain molecules in their blood. Beyond the urgent need for new therapies, one advantage of studying HD is that genetic testing can establish with 100% accuracy whether an individual carries the mutant gene or not, enabling us to study the disease before an individual develops any symptoms. In doing this, we have discovered that the increased inflammation in HD is observed many years before symptom onset. This suggests that inflammation might have long-term effects that are important in the development of the disease.

We have also discovered that inflammation in HD is probably due to the aberrant behaviour of a particular group of immune cells, called monocytes and macrophages. These cells are hyper-responsive to immune stimulation and produce excessive levels of the inflammatory molecules detected in the blood of HD patients. It is becoming clear that the immune system causes changes in the brain and other tissues. Hence, it is conceivable that elevated inflammation in HD patients might be contributing to some of the changes in the brain and/or other tissues that cause the symptoms of the disease to worsen. There are already safe treatments available that will reduce inflammation in humans, but this strategy has not yet been tried in HD.

Our aims are to determine whether inflammation really does contribute to HD progression and if so, whether we can use specific therapeutic agents to lower the inflammation to help treat the disease. We also want to discover the exact means by which the mutant HTT protein causes monocytes and macrophages to behave aberrantly, which may help identify alternative targets for developing treatments for HD.

To achieve these aims, we will undertake a range of experiments in cells from HD patients and in mouse models. We will determine exactly which genes are altered in HD monocytes and macrophages, and will attempt to discover how mutant HTT causes these changes to occur. We will treat HD mice with various agents that are expected to increase or decrease their levels of inflammation, including drugs that reduce inflammation and are already used for this purpose to treat other diseases. We hope that any positive results from these studies will lead to clinical trials in HD patients.

Technical Summary

Huntington's disease (HD) is a fatal, inherited neurodegenerative disorder caused by a CAG repeat expansion in the huntingtin (HTT) gene, leading to an expanded poly-glutamine tract in the HTT protein. Considerable evidence suggests that HD is associated with increased systemic inflammation that may contribute to progression of the disease. We have shown previously that myeloid cells from HD patients are hyper-responsive to stimulation and that this is due to the intrinsic expression of the mutant HTT protein in these cells.

Our hypothesis is that mHTT primes myeloid cell hyper-reactivity to immune stimulation and that the resultant low-level systemic inflammation contributes to HD progression. We aim to determine the mechanisms by which mHTT affects myeloid cells isolated from the blood of HD patients as compared to those from non-HD individuals, by means of full-transcriptome sequencing and methods to monitor transcription factor activation and binding.

We also aim to establish the extent to which components of the peripheral innate immune system contribute to HD and to determine whether systemic inflammation can be targeted to modify disease progression. We will specifically target cells and pathways by genetic and/or pharmacological means in ways that might ameliorate aspects of disease, allowing us to dissect the contribution of specific cell populations, signalling pathways and molecules to HD progression. We hope this will form the basis of further work seeking therapeutic intervention that targets in peripheral innate immune system and systemic inflammation in HD.

Understanding the contribution that systemic inflammation makes to pathogenesis is essential in determining whether targeting it might be a viable therapeutic strategy in HD. A complete mechanistic understanding of these events may identify targets that lead to the development of novel therapies.

Planned Impact

The potential beneficiaries of our research include Huntington's disease patients and families, scientists and clinicians working on HD and/or neuroinflammation, as well as biotechnology and pharmaceutical companies with research and development and/or commercial interests in these areas.

For researchers, an improved understanding of neuroinflammatory events will aid all researchers with an interest in the pathogenic basis of HD. It will also have important implications for the potential development a new therapeutic strategy in HD. To that end, if results are positive, we expect to receive the active support of the CHDI Foundation (whose letter of support is enclosed), a not-for-profit research organisation that works with scientists internationally to discover therapies that slow the progression of HD. The discovery of such therapies will be of considerable commercial interest to companies working in this area.

For patients, we hope the work will form the basis of developing treatments for those who have developed or are likely to develop the disease. HD is a devastating disease that places an intolerable burden on those who suffer from the disorder and their families. The inherited nature of the disease means that family members who act as carers may be doing so with the absolute knowledge that they themselves are going to develop the disease later in life. There are currently no disease-modifying treatments for HD. Significant breakthroughs in our understanding of the pathogenesis of the disease conveys hope to patients and families, and will encourage participation in clinical trials. Understanding the mechanistic underpinnings of the disease will assist in the development of treatments for the benefit of patients.

More widely, the discovery of underlying pathogenic processes and/or therapeutic targets in one neurodegenerative disorder offers hope for researchers working on and patients affected by many others. Neurodegenerative disease is having a serious and long-term impact on an aging UK population, with effects extending from the immeasurable burden on the lives of those affected, to the wider social and economic implications of long-term care. Studies estimate that neurodegenerative disease already costs the UK economy more than cancer and heart disease combined. Due to the likely irreversible nature of the neuronal loss during neurodegeneration, therapies need to target the pre-degenerative disease state. Research in HD, for which there is predictive genetic test that can establish with absolute certainty whether a person will develop the disease later in life, may form the basis of the development of preventative treatments in other neurodegenerative disorders.

Finally, this grant will provide Ralph Andre with the resources to capitalise on his work to date on neuroinflammation as a modifier of disease in HD. He has made a large contribution to the preparation of this grant application; this funding will allow him to build a highly competitive CV from which he can launch his independent career. It will benefit the field by ensuring that a highly capable young investigator is committed to work on HD for the foreseeable future.

Publications

10 25 50
 
Description EXPLORING THE ROLE AND THERAPEUTIC POTENTIAL OF RAB GTPASES IN HUNTINGTON'S DISEASE
Amount £759,052 (GBP)
Funding ID MR/R011621/1 
Organisation Medical Research Council (MRC) 
Sector Public
Country United Kingdom
Start 03/2018 
End 02/2021
 
Description High Content Screening (HCS) platform for HD drug discovery
Amount £381,185 (GBP)
Organisation Takeda Pharmaceutical Company 
Sector Private
Country Japan
Start 12/2017 
End 12/2019
 
Title Patient-derived cell lines 
Description Fibroblasts, lymphoblasts and stem cells 
Type Of Material Biological samples 
Year Produced 2017 
Provided To Others? No  
Impact Ongoing research into genetic modifiers and inflammation in HD 
 
Title RNA sequencing 
Description Primary human monocyte RNAseq from HD patients. RNAseq performed by deCODE, available within the NeurOmics consortium. 
Type Of Material Database/Collection of data 
Year Produced 2016 
Provided To Others? Yes  
Impact Published in Human Molecular Genetics 
 
Description Andreas Weiss 
Organisation Evotec
Country Germany 
Sector Private 
PI Contribution Intellectual contribution and reagents
Collaborator Contribution Intellectual contribution and reagents
Impact None as yet
Start Year 2011
 
Description Dr Paul Lavender 
Organisation King's College London
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual contribution and reagents
Collaborator Contribution Intellectual contribution and reagents
Impact None as yet
Start Year 2014
 
Description Dr Vincent Plagnol 
Organisation University College London
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual contribution and data analyis
Collaborator Contribution Intellectual contribution and data analysis
Impact None as yet
Start Year 2015
 
Description Professor David Greaves 
Organisation University of Oxford
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual contribution
Collaborator Contribution Intellectual contribution and reagents
Impact None as yet
Start Year 2014
 
Description Professor Flaviano Giorgini 
Organisation University of Leicester
Department Department of Genetics
Country United Kingdom 
Sector Academic/University 
PI Contribution Co-application for funding
Collaborator Contribution Co-application for funding
Impact Award of funding, MRC:Research Grant EXPLORING THE ROLE AND THERAPEUTIC POTENTIAL OF RAB GTPASES IN HUNTINGTON'S DISEASE Professor Flaviano Giorgini:University of Leicester MR/R011621/1
Start Year 2018
 
Description Professor Gillian Bates 
Organisation King's College London
Department School of Medicine KCL
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual Contribution and Reagents. Running in vivo aspects of the project.
Collaborator Contribution I was co-applicant on this grant. As well as funding there was intellectual contribution and reagents
Impact Gill Bates - she has been collaborating with Gill Bates since 2000 and is co-applicant on another MRC grant with Gill Bates: 3 papers from 2006 are: Tsang T, Woodman B, McGloughlin G, Griffin J, Tabrizi SJ, Bates GP, Holmes E. Metabanomic Characterisation of the R6/2 Transgenic Mouse Model of Huntington's Disease by High-Resolution MAS 1H NMR spectroscopy. Journal of Proteome Research. 2006 Mar;5(3):483-92. Alexandra Zourlidou, Tali Gidalevitz , Mark Kristiansen , Ben Woodman, Dominic J. Wells, David S. Latchman, Jackie de Belleroche, S. J. Tabrizi, Richard I. Morimoto and Gillian P. Bates. Hsp27 overexpression in the R6/2 mouse model of Huntington's Disease: Chronic Neurodegeneration does not Induce Hsp27 Activation. Human Molecular Genetics. Epub 2007 Mar 14. 2007 May 1;16(9):1078-90. Annette Dalrymple, Edward J Wild, Richard Joubert, Kirupa Sathasivam, Maria Björkqvist, Åsa Petersén, Jeremy Isaacs, Mark Kristiansen, Gillian P Bates Blair R Leavitt, Geoff Keir, Malcolm Ward, S. J Tabrizi. Proteomic profiling of plasma in Huntington's disease reveals neuroinflammatory activation and candidate biomarkers Journal of Proteome Research. 2007 Jul;6(7):2833-40. 2008 -2011 Identification and Cross-validation of Early Stage Phenotypes in Mouse Models of Huntington's disease. Ref G0800846 - MRC - Models of Disease grant. Co-applicant with Professor Gillian Bates (PI) and Dr Michael Modeo (co-applicant). £910,736
Start Year 2008
 
Description Professor Lesley Jones 
Organisation Cardiff University
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual contribution
Collaborator Contribution Intellectual contribution and reagents
Impact None as yet
Start Year 2015
 
Description Professor Neil Aronin 
Organisation University of Massachusetts
Country United States 
Sector Academic/University 
PI Contribution Intellectual contribution and reagents
Collaborator Contribution Intellectual contribution and reagents
Impact Träger U, Andre R, Lahiri N, Magnusson-Lind A, Weiss A, Grueninger S, McKinnon C, Sirinathsinghji E, Kahlon S, Pfister EL, Moser R, Hummerich H, Antoniou M, Bates GP, Luthi-Carter R, Lowdell MW, Björkqvist M, Ostroff GR, Aronin N, Tabrizi SJ. HTT-lowering reverses Huntington's disease immune dysfunction caused by NF?B pathway dysregulation. Brain. 2014 Mar; 137 (Pt 3):819-33.
Start Year 2012
 
Description Professor Peter Holmanns 
Organisation Cardiff University
Country United Kingdom 
Sector Academic/University 
PI Contribution Intellectual contribution and reagents
Collaborator Contribution Intellectual contribution and reagents
Impact None as yet
Start Year 2015
 
Description 18th International Congress of Parkinson's Disease and Movement Disorders 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Participants in your research and patient groups
Results and Impact Invitations and talks of this nature serve to enhance engagement with my research

Various
Year(s) Of Engagement Activity 2014
 
Description 1st Congress of the EAN, Berlin 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Talk on HD research presented to students, scientists and healthcare practitioners.
Year(s) Of Engagement Activity 2015
 
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 25th International Symposium on ALS/MND, Brussels 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented educational talk on HD to MNDA members
Year(s) Of Engagement Activity 2014
 
Description Annual Penney Lecture, Massachusetts General Hospital, Harvard 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented prestigious Jack Penney Lecture sparking questions from a range of students and scientists
Year(s) Of Engagement Activity 2016
 
Description Article/interview in the Telegraph regarding compensation 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 Public/other audiences
Results and Impact Interview and article in the Telegraph re HD compensation paper. Served to expose research to wider audience.

Klöppel S1, Gregory S2, Scheller E3, Minkova L3, Razi A4, Durr A5, Roos RA6, Leavitt BR7, Papoutsi M8, Landwehrmeyer GB9, Reilmann R10, Borowsky B11, Johnson H12, Mills JA13, Owen G8, Stout J14, Scahill RI8, Long JD15, Rees G16, Tabrizi SJ8; Track-On investigators. Compensation in Preclinical Huntington's Disease: Evidence From the Track-On HD Study. EBioMedicine. 2015 Aug 4;2(10):1420-9. doi: 10.1016/j.ebiom.2015.08.002. eCollection 2015.
Year(s) Of Engagement Activity 2015
URL http://hdresearch.ucl.ac.uk/wp-content/uploads/Telegraph-Article.jpg
 
Description Brains Talk Seminar at Roche 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Presented seminar on Hutington's Disease to Roche Pharmaceuticals to aid company strategic planning
Year(s) Of Engagement Activity 2015
 
Description CAG Triplet Repeat Disorders, Tuscany, Italy 
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 on HD research to scientific peers and healthcare professionals
Year(s) Of Engagement Activity 2015
 
Description CHDI 10th Annual HD Therapeutics Conference 
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 on HD studies to researchers, healthcare practitioners and students.
Year(s) Of Engagement Activity 2015
 
Description Chair of clinical trial session at CHDI 11 Annual Conference 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Study participants or study members
Results and Impact Chaired clinical trial blitz session which sparked debates and questions from audience which included students, charitable donrs and industry representatives
Year(s) Of Engagement Activity 2016
 
Description Clinical Neurosciences Alumni Day, Royal Free Hospital1 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Professional Practitioners
Results and Impact Presented educational course to healthcare professionals.
Year(s) Of Engagement Activity 2015
 
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 EHDN 2014 Plenary Meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Participants in your research and patient groups
Results and Impact Invitations and talks of this nature serve to enhance engagement with my research

Various
Year(s) Of Engagement Activity 2014
 
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 HD Website 
Form Of Engagement Activity A magazine, newsletter or online publication
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Articles of this nature serve to enhance engagement with my research

Positive feedback from patients and healthcare professionals
Year(s) Of Engagement Activity 2006,2007,2008,2009,2010,2011,2012,2013,2014,2015,2016
 
Description HD2014: The Milton Wexler Celebration of Life Symposium. 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Engagements and talks of this nature serve to enhance engagement with my research

Various
Year(s) Of Engagement Activity 2014
 
Description Huntington Study Group Presentation, Tampa 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Other audiences
Results and Impact Presented education talk on ongoing clinical trials to HD researchers and clinicians, industry representatives, and advocacy organizations. Talk generated many questions and interest. Aided study/trial planning.
Year(s) Of Engagement Activity 2015
 
Description Huntington's Disease Association's HD Certified Course Programme 
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 on Neurology in Huntington's Disease to health care professionals working in Huntington's Course as part of HDA Certified Course
Year(s) Of Engagement Activity 2016
 
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 Ionis Trial press release 
Form Of Engagement Activity A magazine, newsletter or online publication
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Press release regarding the commencement of the IONIS-HTT-Rx trial. Resulted in media coverage from the BBC, The Times and other national and international publishers and broadcasters.
Year(s) Of Engagement Activity 2015
URL http://www.bbc.co.uk/news/health-34552041
 
Description Key Advances in Neurodegenerative Disorders Meeting, Royal Society of Medicine 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Presented education talk on HD to healthcare professionals
Year(s) Of Engagement Activity 2014
 
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 Lecture at the Young European Scientists meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Undergraduate students
Results and Impact Approximately 400 young scientists from across Europe at a conference to discuss a wide range of topics in the biological and medical sciences
Year(s) Of Engagement Activity 2017
 
Description MRC Centre for Neuromuscular Diseases Seminar Series 2015 
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 on therapeutic strategies in HD to students and professional practitioners alike. The talk generated lot's of interest and questions.
Year(s) Of Engagement Activity 2015
 
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 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 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 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 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 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 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 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:
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 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 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 RSM Medical Genetics Talk 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Professional Practitioners
Results and Impact Presented educational talk as part of course for healthcare professionals.
Year(s) Of Engagement Activity 2015
 
Description Research open day held at the UCLH 
Form Of Engagement Activity Participation in an open day or visit at my research institution
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Public/other audiences
Results and Impact The Tabrizi stall attracted a good number of visitors.

The day was briefly mentioned in the Guardian on-line
Year(s) Of Engagement Activity 2014
 
Description Royal College of Psychiatrists International Congress 2015 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Presented education lecture on 'What have we learnt about compensatory neural networks from large HD cohort studies' to healthcare practitioners
Year(s) Of Engagement Activity 2015
 
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 Teaching to UCL MSc students 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach Local
Primary Audience Postgraduate students
Results and Impact Teaching to UCL post-graduate students
Year(s) Of Engagement Activity 2015,2016
 
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