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Genetic Therapy Accelerator Centre听

The Centre is based at Queen Square and directed by Professor Francesco Muntoni, a Paediatric Neurologist with dual appointments in both institutions (IoN QS and GOSH ICH).

In 2022, Professor Muntoni was made director of the Genetic Therapy Accelerator Centre, a听new gene therapy translational research partnership, based at Queen Square Institute of Neurology and in close collaboration with the 麻豆传媒视频网站 Great Ormond Street Institute of Child Health. This cross-faculty collaboration within 麻豆传媒视频网站 will drive forward an exciting evolving area with direct therapeutic benefits for patients with disabling neurological conditions.听In the last few years, he obtained several international awards, including the 2022 Ottorino Rossi Award, the World Duchenne Organisation 2022 Leadership Awards, the 2023 European Paediatric Neurology Society Jean Aicardi Award and the 2024听Richard Attenborough Award for Outstanding Achievement for听Muscular Dystrophy.

Contacts

Professor Francesco Muntoni, Director of听麻豆传媒视频网站 Genetic Therapy Accelerator Centre, Institute of Neurology, Professor of Paediatric Neurologist, Director of the Dubowitz Neuromuscular Centre,听麻豆传媒视频网站 Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK. Email: f.muntoni@ucl.ac.uk

,听Centre Officer,听Genetic Therapy Accelerator Centre. Email: s.ostler@ucl.ac.uk

,听Professor of Genetic Medicine, Dubowitz Neuromuscular Centre and Genetic Therapy Accelerator Centre. Email: haiyan.zhou@ucl.ac.uk

Professor Jenny Morgan,听Emeritus Professor of Cell Biology,听Dubowitz Neuromuscular Centre and Genetic Therapy Accelerator Centre. Email:听jennifer.morgan@ucl.ac.uk

, Professorial Research Fellow,听Genetic Therapy Accelerator Centre.听Email: j.ng@ucl.ac.uk

Dr Silvia Torelli, Senior Cell Biologist, Dubowitz Neuromuscular Centre and Genetic Therapy Accelerator Centre. Email:听s.torelli@ucl.ac.uk

Dr Sara听Aguti,听Senior Research Fellow, (currently on maternity leave) Genetic Therapy Accelerator Centre.听Email: sara.aguti@ucl.ac.uk

, Senior Research Fellow, (maternity cover),听Genetic Therapy Accelerator Centre.听Email: riccardo.privolizzi.13@ucl.ac.uk

TBC, Senior Research Fellow, Cellular Myologist, Genetic Therapy Accelerator Centre and Dubowitz Neuromuscular Centre.听Email:

, Research Fellow,听Genetic Therapy Accelerator Centre.听Email: l.masjoan@ucl.ac.uk

Research Fellow, Genetic Therapy Accelerator Centre. Email: rebeca.gil@ucl.ac.uk

听Research Assistant and PhD Candidate, Genetic Therapy Accelerator Centre.听Email hester.chu.16@ucl.ac.uk

, Research Assistant,听Genetic Therapy Accelerator Centre.听Email: a.dehkaei@ucl.ac.uk

Camila Vallve Maine, Research Assistant, Genetic Therapy Accelerator Centre. Email: c.maine@ucl.ac.uk

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麻豆传媒视频网站 is ranked number two in London for Life Sciences and number nine globally (QS World University Rankings 2023). 麻豆传媒视频网站 has also been ranked number two in the UK for research power in the latest REF. The faculty is home to a vibrant community of outstanding students, researchers and academics, supported by a committed professional services team who together research, teach and study the science and biology of life.

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Centre Director, Professor Francesco听Muntoni

Professor Francesco Muntoni听is the Centre Director, Professor of Paediatric Neurologist and the Director of the Dubowitz Neuromuscular Centre, at the 麻豆传媒视频网站 Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK. In the Institute and hospital, he led the Novel Therapies Programme of the Biomedical Research Centre听between 2008 and 2022 and also led the Developmental Neuroscience Programme between 2008 and 2018.听Since 2008 he is the co-director of the MRC Centre for Neuromuscular Diseases at 麻豆传媒视频网站.听

Professor Muntoni听has an interest in pathogenesis, deep phenotyping, gene identification for rare neuromuscular conditions and translational research in Duchenne muscular dystrophy, spinal muscular atrophy and congenital myopathies. He is involved in several natural history studies and clinical trials. His research funded by the Department of Health, MRC and the European Commission led to the development and early clinical trials of听2 morpholino antisense oligonucleotides, now approved by the FDA, that induces partial correction of the processing defect of the DMD gene in boys with Duchenne muscular dystrophy. He is the UK chief investigator for multiple clinical trials on DMD and SMA, including genetic therapies involving antisense oligonucleotides and AAV-delivered transgenes.听In the last few years he obtained several international awards, including the 2022 Ottorino Rossi Award, the World Duchenne Organisation 2022 Leadership Awards, the 2023 European Paediatric Neurology Society Jean Aicardi Award听and the 2024听Richard Attenborough Award for Outstanding Achievement for听Muscular Dystrophy.

Email:听f.muntoni@ucl.ac.uk

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Centre Officer, Sophie Ostler: Sophie works as part of the team led by Professor Francesco Muntoni, she is responsible for the day-to-day management of the centre as well as ensuring effective communication practices across the research team, collaborators and funders. In addition to听working closely with the听team and strategic partners, her responsibility focuses on delivering the communications plan,听monitoring the project's expenditure, budgets听and liaising with relevant partners and advisors on a range of research activities.听Sophie studied at the University of Cambridge and has a keen interest in actively promoting and supporting awareness for individuals living and working with rare diseases. She also provides greater understanding听for mental health support, (equality, diversity, Inclusion and听well-being) to staff and students through听outreach programmes and networking.听Email:听s.ostler@ucl.ac.uk

Professor of Genetic听Medicine, Professor Haiyan Zhou听: is Professor of Genetic听Medicine at University College London. Her research focuses on preclinical development of RNA-targeted novel therapies and translational biomedical research in rare genetic disorders. Professor听Zhou is the deputy lead of the Novel Therapy Theme in the NIHR Biomedical Research Centre at 麻豆传媒视频网站 Great Ormond Street Hospital and Institute of Child Health. She is also the director of the MSc Personalized Medicine and Novel Therapies Programme at 麻豆传媒视频网站. Haiyan听is member of World Muscle Society, Oligonucleotide Therapeutic Society and American Society of Gene and Cell Therapy. She is also the UK representative and management committee member of the European Cooperation in Science and Technology (e-COST) on 鈥楧elivery ofAntisense RNA Therapeutics鈥�. Professor Zhou听is the 2021 Harrington UK Rare Disease Scholar and holds a medical degree in clinical medicine and PhD award from Peking Union Medical College. Twitter: @lab_zhou
Emeritus Professor of Cell Biology, Professor Jenny Morgan: Professor Jenny Morgan, research focuses on the biology of skeletal muscle and its capacity to regenerate, using听in vitro听models of stem cell activation, proliferation and differentiation and听in vivo听transplantation models. The major areas of 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.听 Other research areas include the restoration of dystrophin in Duchenne muscular dystrophy. Dubowitz Neuromuscular Centre, Developmental Neurosciences Research and Teaching Department, 麻豆传媒视频网站 Great Ormond Street Institute of Child Health. Email:听jennifer.morgan@ucl.ac.uk听Twitter:听@JenniferEMorgan
Professorial Research Fellow, Professor Jo Ng: 听is a Professorial Research Fellow听developing gene therapies for untreatable neurological disorders that have rapid potential for translation. By understanding the clinical disease and disease pathophysiological mechanisms Dr Ng and her team use Adeno-associated virus (AAV) technology to develop gene therapy approaches for neurological disorders. We evaluate viral capsids, synthetic promoters, and response elements to custom-design gene therapies in order to improve delivery to the CNS and develop cell-specific targeting. To facilitate translation, we use techniques that include lentiviral gene therapy听in vitro听on iPSC-derived neurons from patients with AAV gene therapy in transgenic neurological mouse models to establish听in vivo听gene therapy efficacy and model clinical application. Dr Ng collaborates with academic research groups and industry partners to accelerate translation of new CNS genetic therapy approaches. Email: j.ng@ucl.ac.uk Twitter:听@DrJo_Ng听Dr Silvia Torelli听worked as a senior cell biologist听at the University of Cagliari, Italy听and as a听researcher for 16 years, before joining听the Neuromuscular Unit (Department of Paediatrics) at Hammersmith Hospital Campus, Imperial College London in 2007. In 2007 Silvia听moved to 麻豆传媒视频网站,听Great Ormond Street Institute of Child Health, and听has been working at the Dubowitz Neuromuscular Centre. Her research focuses on understanding the cause of congenital muscular dystrophies and in particular a group of diseases known as 鈥渟econdary dystroglycanopathies' (characterised by the altered glycosylation of alpha-dystroglycan). She also collaborates in studies on Duchenne and Becker Muscular Dystrophies and is specifically involved in the optimisation of techniques for dystrophin quantification.听Email:听s.torelli@ucl.ac.uk

Senior Cell Bilogist, Dr Silvia Torelli: Dr Silvia Torelli听worked as a senior cell biologist听at the University of Cagliari, Italy听and as a听researcher for 16 years, before joining听the Neuromuscular Unit (Department of Paediatrics) at Hammersmith Hospital Campus, Imperial College London in 2007. In 2007 Silvia听moved to 麻豆传媒视频网站,听Great Ormond Street Institute of Child Health, and听has been working at the Dubowitz Neuromuscular Centre. Her research focuses on understanding the cause of congenital muscular dystrophies and in particular a group of diseases known as 鈥渟econdary dystroglycanopathies' (characterised by the altered glycosylation of alpha-dystroglycan). She also collaborates in studies on Duchenne and Becker Muscular Dystrophies and is specifically involved in the optimisation of techniques for dystrophin quantification.听Email:听s.torelli@ucl.ac.uk
Senior Research Fellow,听Dr Sara Aguti (currently on maternity leave): Dr Sara听Aguti听developed an interest in finding successful treatments for neuromuscular and neurological diseases during her听MSc at the University of Siena, Italy, She completed her PhD听under the supervision of Prof. Francesco Muntoni and Prof. Haiyan Zhou and听focused听on developing RNA therapies for collagen VI-related myopathy. Sara听discovered antisense oligonucleotide (ASO) sequences able to correct common mutations in dermal fibroblasts established from collagen VI patients.听After her postdoc, she听moved to MiNA Therapeutics to explore another aspect of RNA therapy: RNA activation by directly targeting DNA and was recently appointed as a senior research fellow at the Genetic Therapy Accelerator Centre,听focusing on developing RNA therapies using different strategies, such as ASOs for splicing modulation or silencing approaches, siRNA, and saRNA, to correct mutations in both neurological and neuromuscular disorders. Additionally, Sara is interested in enhancing the delivery of RNA therapies to specific tissue or cell lines, for one of the main bottlenecks for the development of RNA therapies. Email:听sara.aguti@ucl.ac.uk

Senior Research Fellow, Dr Riccardo听Privolizzi听(maternity cover): is a Senior Research Fellow in the Genetic Therapy Accelerator Centre at the 麻豆传媒视频网站 Queen Square Institute of Neurology. He's also an Honorary Research Fellow and Visiting Lecturer in Gene Therapy at the 麻豆传媒视频网站 EGA Institute for Women's Health and GOS Institute of Child Health, and an ECR Board member of the British Society for Gene and Cell Therapy. Riccardo specialised in the characterisation of novel synthetic promoters and translational AAV gene therapy of a mouse model of infantile parkinsonism-dystonia. Riccardo has a keen interest in the identification and development of novel gene regulatory elements for selective targeting of specific cell types, and is passionate about teaching and science communication. Email:听riccardo.privolizzi.13@ucl.ac.uk
Senior Research Fellow, Cellular Myologist听: Coming soon
Research Fellow, Dr Laia Torres Masjoan: 听obtained her PhD in Neuroscience at King's College London, where she worked on direct neuronal reprogramming, an experimental approach for neuronal regeneration based on the forced expression of key neurogenic factors for the direct conversion of non-neuronal cells (i.e. glial cells) into induced neurons.听听In 2023 she听joined the GTAC team as a Research Fellow to work on the TransNAT project, directed by Professor Muntoni and Professor Zhou.听Her motivation to move her career towards translational research led Laia to be part of the team where she will be testing the efficacy of potentially therapeutic antisense oligonucleotides on in vitro and in vivo models of Duchene Muscular Dystrophy. 听Laia's听work听aims to听optimize听the efficacy, safety and delivery of exon-skipping antisense oligonucleotides (ASOs) for the treatment of the neuromuscular disorder Duchenne Muscular Dystrophy (DMD). Some of DMD are caused by exon deletions altering the reading frame of the dystrophin gene (DMD), and, although four exon-skipping ASOs for the treatment of DMD have been approved by the US FDA, their treatment efficacy remains low. Laia's work at GTAC involves the use of different models of human DMD skeletal and cardiac muscle cells, including hiPSCs-derived and immortalized cells, to test new generation exon-skipping ASOs with different chemical modifications and conjugations to different moieties, and to analyse their exon-skipping efficacy, dystrophin protein restoration, and intracellular trafficking. These analyses will allow the identification of the best candidates for further tests on DMD mouse models.听Email: i.masjoan@ucl.ac.uk

Research Fellow, Dr Rebeca Gil: is a research fellow at GTAC working on gene therapies for neurological disorders with no current effective treatment. She completed her PhD at Royal Holloway University of London focused on gene therapies to treat Duchenne Muscular Dystrophy. She received her MSc on "Genes, drugs and stem cells - novel therapies" at Imperial College London. She studied Biotechnology Engineering for her undergrad, where she developed an interest in novel therapies to treat diseases that had no previous cure. She is currently an early career representative on the board of the British Society for Gene and Cell Therapies (BSGCT). She听has interest in supporting translation of novel therapies, spreading the word on gene & cell therapies to the public and helping early career researchers to get involved in the field; in alignment with her role as BSGCT Early Career Development & Collaboration Subcommittee co-chair and chair of the BSGCT Promotions and Communication Subcommittee..听 Email:听rebeca.gil@ucl.ac.uk; Research Assistant,听Arta Aghaeipour Dehkari听
Research Assistant and PhD Candidate , Hester Chu: is a Research Assistant and PhD Candidate. Coming from a pharmacist background, she is keen to develop novel gene therapy for neurological diseases currently without effective treatments. During her undergraduate, she was awarded the Wellcome Trust Biomedical Vacation Scholarship, and the BSGCT Undergraduate Research Bursary, to conduct adeno-associated virus (AAV) projects at 麻豆传媒视频网站. She is also interested in improving gene therapy efficacy and safety profiles听by regulatory element optimisation and other methods. Email听hester.chu.16@ucl.ac.uk听Twitter:听@HesterChu听

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Research Assistant, Arta Aghaeipour: obtained her BSc in Biomedical Sciences听from Royal Holloway, University of London and an MSc in Human Molecular Genetics from Imperial College London. Her studies at Imperial College听focused听on Human Molecular Genetics coupled with more than three years of dedicated research in nucleic acid therapy and paediatric diseases,听equipped her听with a deep understanding of drug development and disease modelling for rare paediatric disorders.听Arta worked at the Dubowitz Neuromuscular Centre based in 麻豆传媒视频网站 Institute of Child Health, working on Duchenne Muscular Dystrophy and its comorbidities in the brain, as part of the brain Involvement in Dystrophinopathies (BIND) consortium.听In听2023, she joined the GTAC team, directed by Professor听Muntoni and Professor Zhou on screening safe targeted nucleic acid therapies on muscle as a part of the TransNAT consortium. Arta is听passionate about bridging the gap between scientific discoveries and tangible societal improvements. She is particularly drawn to the challenges of understanding disease mechanisms, creating accurate disease models, and developing drugs to enhance the quality of life for patients. The project she is currently working on focuses on developing nucleic acid therapies with better efficacy for Duchenne Muscular Dystrophy as a part of the TransNAT consortium.听Email: a.dehkari@ucl.ac.uk
Research Assistant, Camila Vallve Maine: Camila received a MSci in Applied Biological Research from the Royal Veterinary College, where she undertook three years of a bachelor's degree and one integrated master's year. Her bachelor's thesis was on inflammation markers in genes linked with autism spectrum disorder and her master's work was on adeno-associated virus (AAV) gene therapy for Dravet Syndrome, a rare channelopathy in children.听Currently, she is developing AAV therapies for infantile Parkinsonism.听Camila is passionate about making science more accessible for people of all backgrounds, supporting the Equality, Diversity and Inclusion (EDI) group missions in extending the reach of research, and ultimately wants to improve the lives of children across the globe throughout education and novel genetic therapies development. She is open for collaboration across different sectors, contact her on email for discussion.听Email : c.maine@ucl.ac.uk; 听

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