Antisense nucleic acid splice correction therapy for Duchenne muscular dystrophy and related disorders

Lead Research Organisation: University of Oxford
Department Name: Physiology Anatomy and Genetics

Abstract

Degenerative diseases cause increasing medical and social burdens in aging Western societies. Few of these diseases are treatable and therefore it is vital that new types of therapies are developed. In this research we will study Duchenne muscular dystrophy (DMD), a common, X-linked inherited, degenerative disease of muscle caused by lack of the protein dystrophin, and that is uniformly fatal and currently untreatable, typically leading to the deaths of affected boys in their 20s. We will investigate a new type of gene therapy for DMD, which has already shown promise in two preliminary clinical trials in DMD patients. The therapy, called exon skipping, uses small DNA patches known as antisense oligonucleotides (AOs) to correct the effects of mutations in the dystrophin gene, thereby producing new dystrophin protein of near-normal function and correcting the harmful effects of the disease. The recent clinical trials tested the application of this method in single muscles and therefore one of the major challenges in taking this therapy forward is to develop the means to deliver the AOs effectively to all muscle groups and also to the heart, given that all are affected by the disease. We have recently discovered that attaching small protein fragments known as peptides to the AO allows greatly improved delivery of the AO compounds to multiple muscle groups and heart. In this research we now also plan to test protein fragments that potentially allow the AO to be targeted specifically to muscle and /or heart, initially studying a recently discovered prototype compound known as B-MSP-PMO. We plan to test its long-term effectiveness in two mouse models of DMD, one mild and one severe, and also to attempt to understand better how this compound is targeted to muscle and to what extent it is likely to be safe to use. We also plan to evaluate improved versions of this AO as we are currently working to discover improved peptides that may target the AO to muscle and or heart with even greater efficiency. We will also investigate further recent exciting findings where we have discovered that the delivery of such drugs can be enhanced further by administering them together with a range of sugars. This work will therefore significantly advance the prospects for a disease-modifying therapy for DMD and also for other diseases where exon skipping or AO delivery would be a valuable therapy.

Technical Summary

Experimental nucleic acid based therapies are being intensively investigated as novel strategies for currently untreatable human disease. This Programme aims to develop a detailed molecular understanding of the chemical biological requirements for long-term, targeted, functional and phenotypic antisense oligonucleotide (AO)-mediated splice correction in mouse models of Duchenne muscular dystrophy (DMD). DMD is a fatal muscle degenerative disease and AO mediated splice correction of the DMD pre-mRNA has the potential to treat approximately 75% of DMD patients and has been shown to be well tolerated with therapeutic efficacy in two recent Phase I clinical trials. A critical challenge now in the development of this approach is the need for long-term, targeted, high efficiency systemic correction of the DMD phenotype. In this Programme beginning with state-of-the-art AO-peptide conjugates (B-PMO and B-MSP-PMO) we will study long-term correction of the muscle and cardiac physiological phenotypes following splice correction in dystrophin deficient mdx and dystrophin/utrophin deficient double knockout (DKO) mice. We will also discover and study novel transduction peptides for enhanced AO delivery and splice correction, and develop methods for targeted AO delivery to muscle and cardiac tissues including novel chimeric transduction/targeting peptides and study their mechanisms of action. AO conjugates incorporating these novel peptides will be studied over the long term in DMD animal models. Work will further include study of safety aspects including long term toxicology, tissue biodistribution/pharmacokinetics, and genome-wide off-target and splicing dysregulation effects. Finally, we will further investigate our striking recent findings related to GLUT transporter mediated AO delivery to muscle and cardiac tissues. This is a challenging and integrated programme of work with several streams of study moving from discovery and basic chemistry and chemical biology through to in vitro and in vivo studies in DMD animal models and clinical application via the PI?s participation in the UK MDEX Consortium. It will substantially advance our understanding of the requirements for successful systemic splice correction of the DMD phenotype and will have direct implications for the therapy of related neuromuscular disorders and other diseases where forced manipulation of pre-mRNA splicing and/or targeted AO delivery would be of therapeutic value.

Publications

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A. El Andaloussi S (2012) Use of Cell-Penetrating-Peptides in Oligonucleotide Splice Switching Therapy in Current Gene Therapy

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Aoki Y (2015) Oligonucleotide therapies: the future of amyotrophic lateral sclerosis treatment? in Neurodegenerative disease management

 
Description Action Duchenne
Geographic Reach Europe 
Policy Influence Type Participation in advisory committee
Impact Improved funding environment for new treatments for DMD Discussions wiht regulators re fast track approval of new oligonucleotide therapies for DMD
 
Description EU COST NETWORK WORKSHOPS
Geographic Reach Europe 
Policy Influence Type Participation in advisory committee
Impact IMPROVED REGULATION IN RELATION TO DMD CLINICAL TRIALS
 
Description European COST Award for oligonucleotide therapies
Geographic Reach Europe 
Policy Influence Type Influenced training of practitioners or researchers
Impact Training programme for researchers and collaborative network that has been initiated
 
Description European COST award for exosomes
Geographic Reach Europe 
Policy Influence Type Influenced training of practitioners or researchers
Impact Training programme for researchers and collaborative network that has been initiated
 
Description European training network
Geographic Reach Europe 
Policy Influence Type Influenced training of practitioners or researchers
Impact Recognition that better training of scientists and clinicians and muscle disease biology and therapy is necessary
 
Description IDESC
Geographic Reach Multiple continents/international 
Policy Influence Type Participation in advisory committee
Impact INTERNATIONAL NETWORK TO DISSEMINATE THERAPIES FOR NEUROMUSCULAR DISEASE
 
Description Parkinson's UK Advisory Committee
Geographic Reach UK 
Policy Influence Type Participation in advisory committee
Impact Research funding and research policy
 
Description TREAT NMD
Geographic Reach Europe 
Policy Influence Type Participation in advisory committee
Impact STANDARD OPERATING PROCEDURES AND STANDARDS OF CARE FOR NEUROMUSCULAR DISEASE
 
Description TREAT NMD SYMPOSIUM
Geographic Reach Multiple continents/international 
Policy Influence Type Participation in advisory committee
Impact IMPROVED CLINICAL TRIAL DESIGN
 
Description TREAT-NMD
Geographic Reach Europe 
Policy Influence Type Participation in advisory committee
Impact Clinical trials policy for european neuromuscular disease research
 
Description AFM NETWORK GRANT FOR MD
Amount € 750,000 (EUR)
Organisation French Muscular Dystrophy Association (AFM) 
Sector Charity/Non Profit
Country France
Start 02/2013 
End 08/2015
 
Description Action Duchenne Project Grant
Amount £50,000 (GBP)
Organisation Action Duchenne 
Sector Charity/Non Profit
Country United Kingdom
Start 10/2013 
End 09/2015
 
Description IMI EU AWARD
Amount € 30,000,000 (EUR)
Organisation European Commission 
Sector Public
Country European Union (EU)
Start 11/2012 
End 10/2017
 
Description MD UK DM1
Amount £225,000 (GBP)
Organisation Muscular Dystrophy UK 
Sector Charity/Non Profit
Country United Kingdom
Start 01/2017 
End 12/2019
 
Description MD UK Pip-PMO
Amount £180,000 (GBP)
Organisation Muscular Dystrophy UK 
Sector Charity/Non Profit
Country United Kingdom
Start 08/2016 
End 07/2019
 
Description MD UK studentship KM
Amount £82,173 (GBP)
Funding ID RA3/3031 
Organisation Muscular Dystrophy UK 
Sector Charity/Non Profit
Country United Kingdom
Start 10/2014 
End 09/2017
 
Description MD UK studentship TVW
Amount £110,231 (GBP)
Funding ID RA4/3015/2 
Organisation Muscular Dystrophy UK 
Sector Charity/Non Profit
Country United Kingdom
Start 10/2014 
End 09/2018
 
Description MDC Project Grant
Amount £250,000 (GBP)
Organisation Muscular Dystrophy UK 
Sector Charity/Non Profit
Country United Kingdom
Start 02/2013 
End 01/2017
 
Description MRC BET
Amount £596,000 (GBP)
Organisation Medical Research Council (MRC) 
Sector Academic/University
Country United Kingdom
Start 12/2016 
End 01/2020
 
Description MRC DM1
Amount £883,000 (GBP)
Organisation Medical Research Council (MRC) 
Sector Academic/University
Country United Kingdom
Start 07/2017 
End 06/2020
 
Description MRC DPFS - SMA
Amount £1,008,202 (GBP)
Funding ID MR/L013142/1 
Organisation Medical Research Council (MRC) 
Sector Academic/University
Country United Kingdom
Start 09/2014 
End 09/2017
 
Description MRC PROGRAMME
Amount £1,650,000 (GBP)
Organisation Medical Research Council (MRC) 
Sector Academic/University
Country United Kingdom
Start 01/2011 
End 12/2015
 
Description NOVARTIS FUNDING
Amount £100,000 (GBP)
Organisation Novartis 
Sector Private
Country Global
Start  
 
Description OUI PepGen
Amount £89,000 (GBP)
Organisation University of Oxford 
Department Oxford University Innovation
Sector Private
Country United Kingdom
Start 09/2016 
End 06/2018
 
Description PARKINSONS UK RESEARCH GRANT
Amount £400,000 (GBP)
Organisation Parkinson's UK 
Sector Charity/Non Profit
Country United Kingdom
Start 01/2012 
End 08/2015
 
Description Parkinson's UK Centre Award
Amount £5,000,000 (GBP)
Organisation Parkinson's UK 
Sector Charity/Non Profit
Country United Kingdom
Start 10/2010 
End 09/2015
 
Description PepGen: Peptide platform
Amount £68,000 (GBP)
Organisation Charley's Fund 
Start 06/2017 
End 01/2019
 
Description Pfizer
Amount £1,023,000 (GBP)
Funding ID NA 
Organisation Pfizer Inc 
Sector Private
Country United States
Start 12/2014 
End 11/2017
 
Description RS Inernational Exchange Japan
Amount £12,000 (GBP)
Organisation The Royal Society 
Sector Academic/University
Country United Kingdom
Start 03/2017 
End 02/2019
 
Description SMA Consortium
Amount £1,300,000 (GBP)
Organisation SMA Trust 
Sector Charity/Non Profit
Country United Kingdom
Start 01/2016 
End 09/2019
 
Description WELLCOME TRUST FUNDING
Amount £250,000 (GBP)
Organisation Wellcome Trust 
Sector Charity/Non Profit
Country United Kingdom
Start 10/2011 
End 09/2014
 
Description WELLCOME TRUST FUNDING 2
Amount £150,000 (GBP)
Organisation Wellcome Trust 
Sector Charity/Non Profit
Country United Kingdom
Start 10/2011 
End 09/2014
 
Description WaVe
Amount $380,000 (USD)
Funding ID NA 
Organisation Wave Life Sciences 
Sector Private
Country Unknown
Start 04/2015 
End 09/2016
 
Description Wave DMD
Amount £460,000 (GBP)
Organisation Wave Life Sciences 
Sector Private
Country Unknown
Start 10/2011 
End 07/2019
 
Title EXOSOMES 
Description EXOSOME BASED DRUG DELIVERY VEHICLE 
Type Of Material Technology assay or reagent 
Year Produced 2011 
Provided To Others? Yes  
Impact COMMERCIALISATION 
 
Title EXTRACELLULAR RNA METHODS 
Description IMPROVED ASSAYS FOR EXTRACELLULAR RNAs 
Type Of Material Technology assay or reagent 
Year Produced 2013 
Provided To Others? Yes  
Impact new data in relation to EC RNA new collaborations in relation to EC RNA standardisation of EC RNA protocols 
 
Title Peptides 
Description Cell targeting peptides 
Type Of Material Technology assay or reagent 
Year Produced 2012 
Provided To Others? Yes  
Impact Collaboration and joint research programmes 
 
Title Peptides 
Description Novel cell penetrating peptides 
Type Of Material Technology assay or reagent 
Year Produced 2011 
Provided To Others? Yes  
Impact Collaboration and joint research programmes 
 
Title Synthesis of cell-penetrating peptide morpholino 
Description Cell penetrating peptides are conjugated to neutrally charged morpholino oligonucleotides to improve cellular and tissue uptake 
Type Of Material Technology assay or reagent 
Year Produced 2009 
Provided To Others? Yes  
Impact Utilised for pre-clinical studies for neuromuscular diseases such as Duchenne muscular dystrophy, spinal muscular atrophy and myotonic dystrophy 
 
Title exosome RNA loading 
Description improved methods for insertion of RNA cargoes into extracellular vesicles 
Type Of Material Technology assay or reagent 
Year Produced 2013 
Provided To Others? Yes  
Impact collaboration 
 
Title exosome isolation methods 
Description improved exosome isolation and purification methods 
Type Of Material Technology assay or reagent 
Year Produced 2013 
Provided To Others? Yes  
Impact dramatically improved ability to analyse different extracellular vesicle populations 
 
Description Cardiac aspects of neuromuscular disease 
Organisation Newcastle University
Department Institute of Human Genetics
Country United Kingdom 
Sector Academic/University 
PI Contribution Stiudy of cardiac aspects of neuromuscular disease - expertise and materials
Collaborator Contribution Physiological analysis
Impact n / a
Start Year 2011
 
Description Development of Cell Penetrating Peptides 
Organisation Medical Research Council (MRC)
Department MRC Laboratory of Molecular Biology (LMB)
Country United Kingdom 
Sector Public 
PI Contribution We provide in vivo analysis of novel cell penetrating peptides.
Collaborator Contribution Development of novel cell penetrating peptides for the purposes of delivering antisense oligonucleotides.
Impact Several papers and IPs have resulted from this collaboration in the field of Duchenne muscular dystrophy.
Start Year 2010
 
Description Gait collaboration 
Organisation Medical Research Council (MRC)
Department MRC Laboratory of Molecular Biology (LMB)
Country United Kingdom 
Sector Public 
PI Contribution In vivo work, miRNA work, oligonucleotide work
Collaborator Contribution Materials
Impact 18776238 21062902 20068555 18842625
Start Year 2007
 
Description Karolinska partnership 
Organisation Karolinska Institute
Country Sweden 
Sector Academic/University 
PI Contribution Experimental design and material for transcriptomic and proteomic analysis
Collaborator Contribution Undertaking first stage transcriptomic and proteomic analysis
Impact Roberts et al. Molecular Therapy Nucleic Acids 2012
Start Year 2011
 
Description Leiden University Partnership 
Organisation Leiden University
Country Netherlands 
Sector Academic/University 
PI Contribution Provided materials for study in transgenic mouse model of DMD
Collaborator Contribution In vivo analysis of exon skipping drugs in humanised mouse model of DMD
Impact not yet
Start Year 2012
 
Description MDEX CONSORTIUM 
Organisation University College London
Department Institute of Child Health
Country United Kingdom 
Sector Academic/University 
PI Contribution Pre-clinical evaluation of oligonucleotides for muscular dystrophy therapy
Collaborator Contribution New avenues for experimental work
Impact 19713152
Start Year 2006
 
Description MRC Neuromuscular centre, UCL 
Organisation University College London
Department MRC Centre for Neuromuscular Diseases
Country United Kingdom 
Sector Public 
PI Contribution Therapy development for neuromuscular disease
Collaborator Contribution Clinical material and stem cell expertise
Impact Collaboration and joint research programmes
Start Year 2008
 
Description Muscular dystrophy biology 
Organisation Medical Research Council (MRC)
Department MRC Functional Genomics Unit
Country United Kingdom 
Sector Public 
PI Contribution Expertise and materials
Collaborator Contribution Expertise and materials
Impact Therapy for Duchenne muscular dystrophy: renewed optimism from genetic approaches. Fairclough RJ, Wood MJ, Davies KE. Nat Rev Genet. 2013 Jun;14(6):373-8
Start Year 2012
 
Description Novartis 
Organisation Novartis
Department Institutes for Biomedical Research
Country United States 
Sector Private 
PI Contribution Parkinson's disease expertise
Collaborator Contribution RNA technology
Impact n/a
Start Year 2010
 
Description Oxford Stem Cell Institute 
Organisation University of Oxford
Department Oxford Stem Cell Institute
Country United Kingdom 
Sector Academic/University 
PI Contribution RNA biology / technology
Collaborator Contribution Stem cell expertise and materials
Impact n/a
Start Year 2010
 
Description PHYSICS OXFORD 
Organisation University of Oxford
Department Department of Physics
Country United Kingdom 
Sector Academic/University 
PI Contribution INTELLECTUAL INPUT CELL LINES AND ANIMAL MODELS EXPERIMENTAL DESIGN
Collaborator Contribution NOVEL METHODS OF DNA/OLIGONUCLEOTIDE DELIVERY
Impact WALSH ET AL. ACS NANO 2011
Start Year 2010
 
Description Shire Partnership 
Organisation Shire Pharmaceuticals
Country Ireland 
Sector Private 
PI Contribution Expertise in antisense oligonucleotides for neuromuscular disease
Collaborator Contribution Expertise in oligonucleotide synthesis and toxicology
Impact None yet
Start Year 2012
 
Title EXOSOMES 
Description EXOSOME BASED GENE DELIVERY 
IP Reference WO2010119256 
Protection Patent application published
Year Protection Granted 2010
Licensed No
Impact COMMERCIALISATION
 
Title MIRTRONS 
Description MIRTRON MEDIATED GENE SILENCING 
IP Reference US2011229880 
Protection Patent application published
Year Protection Granted 2011
Licensed No
Impact COMMERCIALISATION
 
Title Peptides 
Description Novel cell penetrating peptides 
IP Reference GB1115014.1 
Protection Patent application published
Year Protection Granted 2011
Licensed No
Impact Funding and joint research programmes
 
Title Peptides 
Description Novel cell penetrating peptides 
IP Reference US20110130346 
Protection Patent application published
Year Protection Granted 2011
Licensed No
Impact Funding and joint research programmes
 
Title Peptides 
Description Novel cell penetrating peptides 
IP Reference WO2009147368 
Protection Patent application published
Year Protection Granted 2009
Licensed No
Impact Funding and joint collaborative research programmes
 
Title DMD oligonucleotide therapy 
Description an exon skipping oligonucleotide product for DMD 
Type Therapeutic Intervention - Cellular and gene therapies
Current Stage Of Development Refinement. Non-clinical
Year Development Stage Completed 2012
Development Status Under active development/distribution
Impact publication IP commercial collaboration 
 
Title SMA oligonucleotide therapy 
Description oligonucleotide exon inclusion therapy for SMA 
Type Therapeutic Intervention - Cellular and gene therapies
Current Stage Of Development Initial development
Year Development Stage Completed 2012
Development Status Under active development/distribution
Impact early preclinical development of a first in class therapuetic agent 
 
Title SMA oligonucleotide therapy 
Description oligonucleotide exon inclusion therapy for SMA 
Type Therapeutic Intervention - Cellular and gene therapies
Current Stage Of Development Initial development
Year Development Stage Completed 2012
Development Status Under active development/distribution
Impact early preclinical development of novel first in class therapeutic 
 
Description A Talk to year 10 students 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach Regional
Primary Audience Schools
Results and Impact A talk to year 10 students interested in a career in medical sciences.
The outcome was pupils from the school asking to conduct work experience in the lab.
Year(s) Of Engagement Activity 2015
 
Description ACTION DUCHENNE MEETING 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Public/other audiences
Results and Impact 150 PE0PLE ATTENDED TALK AND PUBLIC MEETING

REGULAR INPUT TO ORGANISING PATIENT AND FAMILY MEETINGS FOR DUCHENNE PATIENT COMMUNITY TO DISCUSS RESEARCH PROGRESS
Year(s) Of Engagement Activity 2007,2008,2009,2010,2011
 
Description Action Duchenne Charity 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Patients, carers and/or patient groups
Results and Impact Formal presentation on research
Year(s) Of Engagement Activity 2015,2016,2017
 
Description Action Duchenne Conference 2013 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Type Of Presentation Keynote/Invited Speaker
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Public engagement

EDUCATION
Year(s) Of Engagement Activity 2007,2009,2010,2011,2012
 
Description BBC INTERVIEWS 
Form Of Engagement Activity A press release, press conference or response to a media enquiry/interview
Part Of Official Scheme? No
Type Of Presentation Keynote/Invited Speaker
Geographic Reach International
Primary Audience Media (as a channel to the public)
Results and Impact BBC TV INTERVIEW

NUMEROUS OFFERS TO WRITE ABOUT AND TALK ABOUT OUR WORK
Year(s) Of Engagement Activity 2011,2012
 
Description EU COST Network Workshops 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Talk: "Enhancing systemic biodistribution of SSOs using peptide delivery: The next step for SSO treatment of SMA"
Year(s) Of Engagement Activity 2014
 
Description Families of Spinal Muscular Atrophy Conference 2013 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact Poster Presentation "Peptide delivery of splice switching oligonucleotides for the treatment of SMA "
Year(s) Of Engagement Activity 2013
 
Description International congress of myology 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Poster Presentation :Development of a cell-penetrating peptide for the delivery of antisense oligonucleotides to peripheral and CNS tissues of spinal muscular atrophy mice
Year(s) Of Engagement Activity 2016
 
Description MDA conference 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Type Of Presentation Keynote/Invited Speaker
Geographic Reach International
Primary Audience Public/other audiences
Results and Impact public engagement

education and policy
Year(s) Of Engagement Activity 2013
 
Description MDC NEWSLETTER 
Form Of Engagement Activity A magazine, newsletter or online publication
Part Of Official Scheme? Yes
Geographic Reach Regional
Primary Audience Public/other audiences
Results and Impact SHORT NEWS ARTICLE

INVITATIONS TO TALK ABOUT WORK
Year(s) Of Engagement Activity 2010,2011
 
Description MDC Visit 
Form Of Engagement Activity Participation in an open day or visit at my research institution
Part Of Official Scheme? No
Type Of Presentation Workshop Facilitator
Geographic Reach National
Primary Audience Public/other audiences
Results and Impact public engagement

education, engagement, patient recruitment
Year(s) Of Engagement Activity 2011,2013
 
Description Muscular Dystrophy UK 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Patients, carers and/or patient groups
Results and Impact Often invited to visit parent/patient meetings to chat
Year(s) Of Engagement Activity 2014,2015
 
Description Muscular dystrophy campaign public meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Type Of Presentation Keynote/Invited Speaker
Geographic Reach National
Primary Audience Public/other audiences
Results and Impact ~100 people attended a public presentation

Funding
Refining research strategy of organisation
Policy discussion
Year(s) Of Engagement Activity 2012
 
Description Neuroscience School of Advanced Studies (NSAS) forum - Non-coding RNA in Brain Function and Disorders 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Study participants or study members
Results and Impact Neuroscience School of Advanced Studies (NSAS) forum 1 day full teaching of Non-coding RNA in Brain Function and Disorders to course members
Year(s) Of Engagement Activity 2016
 
Description New Directions in Biology and Disease of Skeletal Muscle meeting 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact Spoke at the New Directions in Biology and Disease of Skeletal Muscle meeting in Orlando
Year(s) Of Engagement Activity 2016
 
Description Oligonucleotide Delivery: Biology, Engineering and Development Conference 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Presentation entitled "A new non-base chemical modifier 'ZEN' enhances efficacy of splice-switching oligonucleotides".
Year(s) Of Engagement Activity 2012
URL http://www.eurekalert.org/pub_releases/2012-06/eci-od062712.php
 
Description Oligonucleotide Therapeutics Society 2011 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Poster Presentation "NOVEL CELL PENETRATING PEPTIDES FOR SKELETAL AND CARDIAC MUSCLE DELIVERY OF PMO ANTISENSE OLIGONUCLEOTIDES FOR THE TREATMENT OF DUCHENNE MUSCULAR DYSTROPHY"
Year(s) Of Engagement Activity 2011
 
Description Oligonucleotide Therapeutics Society 2012 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Industry/Business
Results and Impact Poster Presentation: "A new non-base chemical modifier "ZEN" enhances efficacy of splice-switching oligonucleotides"
Year(s) Of Engagement Activity 2012
 
Description Parent Project Muscular Dystrophy's Annual Connect Conference 
Form Of Engagement Activity Participation in an activity, workshop or similar
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Patients, carers and/or patient groups
Results and Impact Attended the Parent Project Muscular Dystrophy's Annual Connect Conference in Orlando
Year(s) Of Engagement Activity 2016
 
Description Plenary Lecture, MDA Scientific Conference, Washington DC 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach International
Primary Audience Professional Practitioners
Results and Impact The 2017 MDA Scientific Conference brings together influential professionals in the academic, government, industrial and clinical arenas who work in various ways to help drive scientific and therapeutic advancements and discoveries. Nearly 450 leaders in the neuromuscular field gather for this prestigious conference to share critical updates on a wide range of scientific topics.
Year(s) Of Engagement Activity 2017
URL https://www.mda.org/conferences/2017-scientific-conference
 
Description Public talk/lecture/debate - DMD 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Public/other audiences
Results and Impact Engage DMD patients, families, carers and the DMD community more generally in thinking about progress in new therapy development and the associcated challenges.
Year(s) Of Engagement Activity 2014
 
Description TREAT-NMD 
Form Of Engagement Activity A formal working group, expert panel or dialogue
Part Of Official Scheme? No
Type Of Presentation Keynote/Invited Speaker
Geographic Reach International
Primary Audience Participants in your research and patient groups
Results and Impact EDUCATION AND POLICY IN RELATION TO NEUROMUSCULAR DISEASE

EDUCATION AND POLICY IN RELATION TO NMD
Year(s) Of Engagement Activity 2009,2011,2013
 
Description TV broadcast in support of BBC Lifeline Appeal 
Form Of Engagement Activity A broadcast e.g. TV/radio/film/podcast (other than news/press)
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Public/other audiences
Results and Impact Sue Barker, President of Muscular Dystrophy UK, talks to Professor Wood on BBC Lifeline, 22 March. Muscular Dystrophy UK supports people
living with these conditions and invests in groundbreaking research.
Year(s) Of Engagement Activity 2015
URL http://www.musculardystrophyuk.org/app/uploads/2015/03/COMM5-B_-_Lifeline_posters_Prof_Matthew_Wood....
 
Description UK Neuromuscular Translational Research 2013 
Form Of Engagement Activity A talk or presentation
Part Of Official Scheme? No
Geographic Reach National
Primary Audience Professional Practitioners
Results and Impact Poster presentation :A new non-base chemical modifier "ZEN" enhances efficacy of splice-switching oligonucleotides
Year(s) Of Engagement Activity 2013