UCL

Known as ‘London’s Global University’, University College London (UCL) employs 4,000 academic and research staff in over 50 departments and institutes. UCL has a global reputation for excellence in research and is committed to delivering impact and innovations that enhance the lives of people in the UK, across Europe and around the world. UCL is consistently placed in the global top 20 across a wide range of university rankings (currently 7th in the QS World University Rankings with a score of 94.6). Furthermore, the Thomson Scientific Citation Index shows that UCL is the 2nd most highly cited European university and 14th in the world.

The coordinating institution, UCL, has extensive experience in managing EU funded projects under FP4, FP5, FP6, FP7 and Horizon 2020. UCL is currently involved in more than 500 EU-funded projects, of which more than 340 are funded through Horizon 2020.  UCL was the top performing university in the first year of Horizon 2020 and 7th institution overall in EU collaborative research (#3 in the UK). UCL has expanded its European Research and Innovation Office (ERIO; http://www.ucl.ac.uk/research-services/euro-funding/erio) as a dedicated central support and project assurance service for all Horizon 2020 projects in which it participates. This service includes a specialist European Project Management section employing eight full-time members of staff, who are successfully managing a total of 30 concurrent FP7 and Horizon 2020 projects (27 of which are Horizon 2020) with a total combined budget of over €155 M. UCL also receives the highest share of any UK university of the UK Government’s strategic investment fund, and has recently invested more than €310 million into state-of-the-art infrastructure to facilitate cutting-edge research across a broad range of disciplines.

Based on a research strategy that is oriented around a series of ambitious “Grand Challenges”, including ‘Human wellbeing’, UCL nurtures thriving and engaged communities of world-leading academics across the entire research and innovation spectrum, from arts and humanities to the basic and applied sciences and healthcare. Complementing its leading research and innovation portfolio, UCL is internationally recognised as a centre of excellence for science communication. This has been recognized through UCL’s selection as one of six national Beacons for Public Engagement in the UK, funded by the UK government to deliver a step change in science outreach and impact activities.”

UCL is the coordinator of the BIND project.

UCL

Website

 www.ucl.ac.uk

Team

Muntoni

Professor Francesco Muntoni

Position
Director of  the Dubowitz Neuromuscular Centre at Great Ormond Street Institute of Child Health and Great Ormond Street hospital

Profile
Prof Francesco Muntoni is the director of the Dubowitz Neuromuscular Centre at Great Ormond Street Institute of Child Health and Great Ormond Street hospital. He leads a large multidisciplinary team involved in the development and implementation of standards of care for neuromuscular disorders in the UK and globally. He identified more than 30 neuromuscular genes and has led a consortium that identified an optimal sequence to induce exon 51 and 53 skipping in DMD patients with eligible deletions. He performed investigator led first in man studies with this antisense oligonucleotide that subsequently became the first FDA approved drug in 2016 (eteplirsen).  He led the FP7 SKIP-NMD EU consortium in which golodirsen, a novel morpholino antisense oligonucleotide targeting exon 53 was developed for DMD.

Role
Professor Muntoni coordinates the multidisciplinary BIND consortium.

Jennifer Morgan

Professor Jennifer Morgan

Position in Organisation
Professor of Cell Biology

Profile
Jennifer Morgan has been a professor at the UCL Great Ormond Street Institute of Child Health since 2014. She is head of the Molecular Neurosciences Section in the Developmental Neurosciences Programme. The main areas of her research are the identification of stem cells that contribute to skeletal muscle regeneration, and the genetic and functional manipulation of these cell populations to enhance muscle repair in skeletal muscle regenerative medicine and in the treatment of muscular dystrophies. She has expertise in mouse models of neuromuscular diseases and dystrophin quantification.

Role in the project
Jennifer Morgan co-leads WP2 with Federica Montanaro and will be contributing to the correlation of the cellular and molecular findings from WP2 with the behavioural outcomes in WP3 and WP4, and in determining the effects of exon skipping in mdx52 mice on dystrophin isoform.

Federica Montanaro

Dr Federica Montanaro

Position in Organisation
Senior Lecturer

Profile
Federica Montanaro is a senior lecturer at Dubowitz Neuromuscular Centre at UCL Great Ormond Street Institute of Child Health. Her translational research focuses on the development of next-generation AAV-micro-dystrophin gene therapy vectors with improved cardio-protective potential. She is an expert in the purification of intact dystrophin protein complexes from tissues followed by the identification of their protein components by mass spectrometry. In addition, she has expertise in mouse models of neuromuscular diseases, in particular the mdx5cv mouse model.

Role in the project
Dr. Montanaro will be directly involved in the following work packages: WP2, WP3 and WP4. She is a co-lead on WP2 with Prof. Jennifer Morgan.

David Skuse

Prof David Skuse

Position in Organisation
Prof of Behavioural and Brain Sciences

Profile
Porfessor Skuse has conducted a range of research with individuals who have disorders on the autism spectrum over the past 20 years, including a number of highlighted studies linking deficits in social cognition to abnormal neuro-physiological responses, and functional brain changes measured by fMRI. He contributed to the evidence base for the DSM-5 revision of the ASD diagnostic framework and published the first independent evaluation supporting new criteria He has published widely on the cognitive and psychiatric consequences of X chromosome aneuploidies, and on genetic risks for social communication disorders.

Role in the project
Prof. Skuse will be involved in the interpretation of neurophysiological measurements in DMD and BMD patients, using a battery of tests (WP5). Prof Skuse will be instrumental in the future links with ERNs and other research networks focused on ASD.

William Mandy

Dr. William Mandy

Position in Organisation
Research Director of UCL Clinical Psychology and Language Sciences

Profile
Dr Mandy’s most important research-related contributions have served to improve knowledge and care of neuro-developmental disorders, in particular autism.
These include: Enhancing conceptualisation and measurement of autism and autistic traits, including the development of new measures; Identifying gender differences in autism, to reduce the current diagnostic bias against girls and women on the autism spectrum;Increasing understanding of the epidemiology of autistic traits; Applying state-of-the-art neuropsychiatric assessment techniques to characterise the neurodevelopmental phenotype of DMD.

Role in the project
Dr Mandy will be involved in neuropsychiatric phenotyping of patients with Duchenne and Becker muscular Dystrophy (WP5).

Christopher Clark

Prof Christopher Clark

Position in Organisation
Professor of Imaging and Biophysics

Profile
Professor Chris Clark is an MRI physicist with a special interest in neuroimaging in neuro-paediatrics using quantitative MRI methods, in particular diffusion MRI. He is currently a Professor at the Great Ormond Street Institute of Child Health, University College London where he leads the Developmental Imaging and Biophysics Section. Professor Clark lead developments in diffusion MRI including the first diffusion maps in human spinal cord in vivo, contributed to the biophysical understanding of the diffusion signal and was amongst the first to demonstrate the potential of tractography for neurosurgical planning. He has worked with Professor Muntoni on the application of diffusion MRI and other MRI methods in brain and muscle in Duchenne Muscular Dystrophy. This includes the BIOIMAGE-NMD project funded by the EU.

Role in the project
Professor Clark will lead the human neuroimaging component at UCL (Work Package 6).

Catherine Moss

Dr Catherine Moss

Position in Organisation
Scientific Coordinator for BIND

Profile
Catherine Moss has a background in biomedical research, and extensive experience in using a range of biophysical and biochemical techniques to study proteases as candidate drug targets in pathogenic parasites, and in human MHC II antigen presenting cells. She has subsequently been involved in research administration and project management of EU funded projects at the University of Oxford and the University of Reading.

Role in the project
Catherine Moss will be part of the Management Support Team and will oversee the scientific aspects of the day-to-day management of BIND.

Eranda Begaj

Eranda Begaj

Position in Organisation
Assistant Project Manager for BIND

Profile
Eranda holds a Bachelor degree in Political Science from the Panteion University of Social and Political Sciences and a diploma in EU Studies from the University Louvain de Catholique, Belgium. Since 2015, she has participated in various projects related to ERASMUS Plus and H2020 programme. Eranda holds the position of Assistant Project Manager at UCL European Research and Innovation Office.

Role in the project
Eranda Begaj will be part of the Management Support Team and will oversee the financial, contractual and administrative aspects of the day-to-day management of BIND (WP1).

David Chapman-Jones

Dr David Chapman-Jones

Position in Organisation
Intellectual Property and Exploitation Manager for BIND

Profile

  • Dr David Chapman-Jones (he/him)
  • Interim Lead for GMP Facility: Gene and Cell GMP Facility:
  • Zayad Centre for Research into Rare Diseases in Children
  • Great Ormond Street Hospital for Children NHS Foundation Trust
  • Innovation and Business Development Manager: Biomedical Research Centre, Division of Research and Innovation
  • Great Ormond Street Hospital for Children NHS Foundation Trust
  • UCL Great Ormond Street Institute of Child Health
    30 Guilford Street | London | WC1N 1EH
    www.gosh.nhs.uk/research-and-innovation
    +44 (0)20 7905 2677 ext. 42712
    david.chapman-jones@gosh.nhs.uk
  • Emeritus Professor of Healthcare, University of the West of Scotland
  • Honorary Research Fellow UCL
  • Science and Medical Doctorates with Masters of Laws in Law and Medical Ethics (Member of the Inner Temple Inns of Court)
  • Degree Names: Doctor of Philosophy (PhD), MD, MSc, LLM (Med), LLB (Legum Baccalaureus) MA (ed)
  • Field of Study Electroceutical Medicine and Gene Expression Manipulation
  • Sports & Exercise and Musculoskeletal Medicine and Wound Care
  • Activities and Societies: Worshipful Society of Apothecaries, Inner Temple Inns of Court, Thoroughbred Racehorse Association, Racehorse Owners Association

Role in the project
Dissemination and Expoitation Manager