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Genetic Therapy Accelerator Centre
The Genetic Therapy Accelerator Centre (GTAC) was established in 2022 to develop cutting-edge RNA, AAV and Cellular Myology therapies for neurological and neuromuscular disorders. The Centre is based at the Institute of Neurology, Queen Square House and is directed by Professor Francesco Muntoni, a Paediatric Neurologist with dual appointments in both institutions at UCL (IoN QS and GOSH ICH). Professor Jo Ng leads the AAV team, working with Senior Fellow, Dr Riccardo Privolizzi. Dr Sara Aguti leads on RNA, and Dr Massimo Ganassi leads on Cellular Myology.
A joint initiative between Queen Square Institute of Neurology and Great Ormond Street Hospital’s Institute of Child Health, the centre fosters collaboration with clinicians and cross-discipline basic scientists to leverage the multidisciplinary expertise and develop the next generation of genetic therapies for clinical translation.
Centre Director, Professor Francesco Muntoni
Professor Francesco Muntoni is Professor of Paediatric Neurology and the Director of the Dubowitz Neuromuscular Centre, at the UCL Great Ormond Street Institute of Child Health and Great Ormond Street Hospital for Children, London, UK.
In the Institute and the hospital, he led the Novel Therapies Programme of the Biomedical Research Centre and between 2008 and 2022 and also the Developmental Neuroscience Programme between 2008 and 2018.
Since 2022 Muntoni is also director of the Genetic Therapy Accelerator Centre, a new gene therapy translational research partnership, based at Queen Square Institute of Neurology and in close collaboration with the UCL Great Ormond Street Institute of Child Health. This cross-faculty collaboration within UCL is driving forward an exciting evolving RNA and AAV gene therapy area with direct therapeutic benefits for patients with disabling neurological conditions.
Muntoni 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 lead to the development and early clinical trials of 2 morpholino antisense oligonucleotides, now approved by FDA, that induce partial correction of the processing defect of the DMD gene in boys with Duchenne muscular dystrophy.
Muntoni is the chief investigator of the European consortium BIND (Brain Involvement in Dystrophinopathies) focusing on the brain comorbidities observed in a proportion of patients with Duchenne muscular dystrophy, and exploring both their biological basis, clinical assessment tools and preclinical translational research.
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, the 2024 Richard Attenborough Award for Outstanding Achievement from Muscular Dystrophy UK and the 2026 Novo Nordisk Prize.
Email: f.muntoni@ucl.ac.uk
Sophie works as part of the team led by Professor Francesco Muntoni, she is responsible for the management of the centre as well as ensuring effective communication practices across the research team, collaborators and funders. In addition to working closely across the university and strategic partners, her responsibility focuses on delivering the communications plan, monitoring grants, project expenditure, and liaising with relevant partners to enhance interdisciplinary collaborative research. Sophie studied Life Sciences at the University of Cambridge, and the University of Edinburgh Medical School, she has a keen interest in actively promoting and supporting awareness for individuals living and working with rare diseases. She also provides support on mental health and well-being to staff and students.
Email: s.ostler@ucl.ac.uk
Professor Haiyan Zhou is Professor of Genetic Medicine at University College London and a UK Harrington Rare Disease Scholar. Her research focuses on the preclinical development and clinical translation of RNA-targeted novel therapies and translational biomedical research for rare genetic disorders. Professor Zhou is the Director and co-lead (alongside Professor Muntoni) the UK Platform of Nucleic Acid Therapy (UPNAT) for rare disease, a specialist rare disease node supported by MRC and NIHR. She serves as the Deputy Lead of the Novel Therapy Theme in the NIHR Biomedical Research Centre at UCL Great Ormond Street Hospital and Institute of Child Health (GOSH BRC) where she leads the RNA therapy program for paediatric rare diseases.
Additionally, Professor Zhou co-leads the Therapeutic Innovation and Trials Community Domain of Genomics England Research Network and is the principal investigator of multiple research projects on RNA therapeutics for a wide range of genetic diseases. Professor Zhou is actively involved in postgraduate education as Co-Director of the MSc Cell, Gene, and Novel Therapies Programme at UCL. Professor Zhou is a member of the World Muscle Society, the Oligonucleotide Therapeutic Society and the American Society of Gene and Cell Therapy. She holds a medical degree in clinical medicine and a PhD from Peking Union Medical College.
Email: h.zhou@ucl.ac.uk x.com: @lab_zhou
Professor Jo Ng is a Professorial Research Fellow developing gene therapies for untreatable neurological disorders that have rapid potential for translation. By understanding the clinical disease and disease pathophysiological mechanisms. Professor 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 in vitro on iPSC-derived neurons from patients with AAV gene therapy in transgenic neurological mouse models to establish in vivo gene therapy efficacy and model clinical application. Professor Ng collaborates with academic research groups and industry partners to accelerate translation of new CNS genetic therapy approaches.
Email: j.ng@ucl.ac.uk x.com: @DrJo_Ng
Dr Silvia Torelli worked as a Senior Cell Biologist at the University of Cagliari, Italy and as a Senior Cell Biresearcher, before joining the Neuromuscular Unit (Department of Paediatrics) at Hammersmith Hospital Campus, Imperial College London in 2007. In 2007 Silvia moved to UCL, Great Ormond Street Institute of Child Health, and 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 “secondary 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.
Email: s.torelli@ucl.ac.uk
Dr Sara Aguti developed an interest in finding successful treatments for neuromuscular and neurological diseases during her MSc at the University of Siena, Italy, She completed her PhD under the supervision of Prof. Francesco Muntoni and Prof. Haiyan Zhou and focused on developing RNA therapies for collagen VI-related myopathy. Sara discovered antisense oligonucleotide (ASO) sequences able to correct common mutations in dermal fibroblasts established from collagen VI patients. After her postdoc, she moved to MiNA Therapeutics to explore another aspect of RNA therapy, RNA activation by directly targeting DNA. Sara was appointed as a senior research fellow at the Genetic Therapy Accelerator Centre (GTAC) in 2023, 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: sara.aguti@ucl.ac.uk
Dr Massimo Ganassi is a Senior Research Fellow in the Genetic Therapy Accelerator Centre (GTAC), IoN and part of the Dubowitz Neuromuscular Centre (DNC), at the UCL ICH, (GOS-ICH), Great Ormond Street. Massimo is also a visiting Senior Research Fellow at the Randall Centre for Cell & Molecular Biophysics, within the Faculty of Life Sciences & Medicine at King’s College London. Massimo holds a degree in Molecular and Regenerative Medicine from University of Modena and Reggio Emilia (Italy), and is interested in understanding the molecular regulation of muscle homeostasis and plasticity, particularly on the formation, maintenance and regeneration of efficient musculature throughout life with the goal of developing effective therapies. He is a member of the British Society for Cell Biology and of the British Society for Developmental Biology. He is editorial board member for the European Journal of Translational Myology and Frontiers in Physiology (Striated Muscle) and Reviewer board member for MDPI journal Muscles. Massimo also co-leads the Skeletal Muscle and Associated Diseases (CLNE0022) teaching module within the Faculty of Brain Sciences.
Email: m.ganassi@ucl.ac.uk
Dr Riccardo Privolizzi is a Senior Research Fellow in the Genetic Therapy Accelerator Centre at the UCL Queen Square Institute of Neurology. He's also an Associate Lecturer in Gene Therapy within the UCL Division of Surgery and Interventional Science and GOS Institute of Child Health, and an Honorary Research Fellow at the UCL EGA Institute for Women’s Health. Riccardo specialised in the characterisation of novel synthetic promoters and translational AAV gene therapy of a mouse model of infantile parkinsonism-dystonia. He 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. Riccardo is also an ECR Board member of the British Society for Gene and Cell Therapy, where he co-chairs the Early Career Development and Collaboration sub-committee and is a mentor for ECRs.
Email: riccardo.privolizzi.13@ucl.ac.uk bsky.social.com: ‪@drrickprivolizzi.bsky.social‬
Dr Margherita Berti is a Research Fellow at the Genetic Therapy Accelerator Centre, UCL Queen Square Institute of Neurology, where she focuses on advancing gene therapy approaches for neuromuscular diseases, such as Collagen-6-deficiency. She previously worked as a Postdoc Researcher at the ClĂnica InvestigaciĂłn MĂ©dica Aplicada (CIMA) in Pamplona, investigating glial cell behaviour in Alzheimer’s and Parkinson’s disease through antisense oligonucleotide-based gene modulation. Margherita completed her PhD in Molecular Medicine at San Raffaele Institute for Gene and Cell Therapy (SR-TIGET), where she developed a murine model for a lysosomal storage disorder and conducted preclinical studies on lentiviral gene therapy. Earlier in her career, during her MSc and initial research training at the UCL Institute for Women’s Health, she engineered AAV vectors for the treatment of Dravet Syndrome, developing a interest in gene therapy and translational neuroscience research.
Email: margherita.berti.17@ucl.ac.uk
Dr Haoyu Chi is a Research Fellow at the Genetic Therapy Accelerator Centre (GTAC), UCL Queen Square Institute of Neurology. He completed his PhD at UCL under the supervision of Professor Michael Duchen, where he studied early metabolic changes linked to neurodegeneration in Down syndrome and APP duplication models of Alzheimer’s disease. His current research project focuses on developing a synthetic protein engineering approach to reassemble midi-dystrophin for AAV-based gene therapy in Duchenne muscular dystrophy (DMD), with the aim of translating this work towards clinical application.
Email: caesar.chi.15@ucl.ac.uk
Dr Ben Jarvis is a Research Fellow in the Genetic Therapy Accelerator Centre (GTAC) at the UCL Queen Square Institute of Neurology. He obtained his PhD under the supervision of Professor Adrian Woolf and Dr Neil Roberts at the University of Manchester where his research involved investigation of novel gene therapy approaches to treating rare early onset lower urinary tract (REOLUT) disorders. His research aims to develop viral vector-mediated therapies for devastating childhood diseases affecting the central and peripheral nervous systems for which current treatments are lacking. Since 2025, he is developing recombinant adeno-associated viral vector (rAAV) strategies to deliver therapeutic transgenes to the nigrostriatal network affected in familial Parkinson’s disease (PD).
Email: b.jarvis@ucl.ac.uk
Dr Ricardo Parolin Schnekenberg, is a Research Fellow at the Genetic Therapy Accelerator Centre, UCL Queen Square, Institute of Neurology. His work focuses on the genetics and mechanisms of rare neurodevelopmental and neurodegenerative disorders. He completed a DPhil in Clinical Neurosciences at the University of Oxford under the supervision of Prof. Andrea Nemeth, where he developed stem cell models to study neurodevelopmental disorders, with a particular emphasis on ITPR1-related conditions. He went on to work as a postdoctoral fellow with Prof. Andrea Cortese, investigating the molecular mechanisms underlying CANVAS (Cerebellar Ataxia, Neuropathy, and Vestibular Areflexia Syndrome).
At the Genetic Therapy Accelerator Centre, Dr Schnekenberg is a clinical research fellow dedicated to understanding disease mechanisms and advancing therapeutic development for ITPR1-related disorders, including SCA29, SCA15, and Gillespie Syndrome. His research bridges clinical insight with laboratory science, aiming to translate genetic discoveries into meaningful treatments for patients.
Email: r.schnekenberg@ucl.ac.uk
Dr Rubing Shi is a Research Fellow in the Department of Neuromuscular Diseases at the UCL Queen Square Institute of Neurology. She obtained her PhD under the supervision of Professor Yongting Wang and Professor Guo-Yuan Yang at Shanghai Jiao Tong University, where her research focused on developing stem cell and gene therapy approaches for cerebrovascular diseases, such as ischemic stroke. Following her interests, Rubing joined GTAC as a Postdoctoral Researcher in 2025 and is developing adeno-associated virus (AAV) based gene therapy strategies for mitochondrial DNA polymerase (POLGÎł)-related primary mitochondrial diseases.
Email: rubing.shi@ucl.ac.uk
Dr Konstantina Tetorou, is a Research Fellow at the Genetic Therapy Accelerator Centre, UCL Queen Square, Institute of Neurology. She holds a PhD in Neuroscience focusing on molecular and inflammatory pathways of neonatal hypoxic-ischaemic encephalopathy and their role in potential new therapeutic approaches. As a postdoctoral researcher, she is involved in molecular and cellular studies of neuromuscular disorders, in particular dystrophinopathies, as well as gene therapy studies. Konstantina has expertise in dystrophin interactome in the brain and in in vivo models of Duchenne muscular dystrophy. Her main research area is the brain involvement in Duchenne muscular dystrophy and studies on translational therapeutic approaches for the disease.
Hester Chu is a Research Assistant and PhD Student. 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 UCL. She is also interested in improving gene therapy efficacy and safety profiles by regulatory element optimisation and other methods.
Email hester.chu.16@ucl.ac.uk x.com: @HesterChu
Eleanor Davis is a Research Assistant and joined the Genetic Therapy Accelerator Centre (GTAC) at IoN. She completed her BSc in Biology at the University of Bristol, followed by an MSc in Cell and Gene Therapy at University College London. Her role focuses on developing translational adeno-associated virus (AAV)-based gene therapy strategies for Duchenne Muscular Dystrophy.
Email: eleanor.davis.24@ucl.ac.uk
Anis Hassan is a Research Assistant at the Genetic Therapy Accelerator Centre (GTAC). She completed her BSc in Neuroscience at the University of Michigan and later pursued an MSc in Biotechnology at the University of Edinburgh. Anis first gained exposure to gene therapy as a research assistant at King’s College London and has continued to build on her passion through her work with GTAC. Her role focuses on facilitating the development of adeno-associated virus (AAV)-based gene therapy strategies for infantile Parkinsonism.
Email: anis.hassan.19@ucl.ac.uk
Camila Maine 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. Currently, she is developing AAV therapies for infantile Parkinsonism. 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.
Email : c.maine@ucl.ac.uk
Le Hong Ngoc Nguyen (Mary) is a research assistant at the Genetic Therapy Accelerator Centre (GTAC), IoN, and UCL ICH (GOS-ICH), Great Ormond Street. Mary completed her BSc in Biomedical Science at King’s College London and MSc in Dementia: Causes, Treatment and Research (Neuroscience) at University College London. Her work focuses on developing antisense oligonucleotides to treat the rare disease Asparagine synthetase deficiency (ASNSD)
Email: le.nguyen.24@ucl.ac.uk
Susanna Rathnayaka is a Research Assistant in the Genetic Therapy Accelerator Centre (GTAC), IoN and part of the Dubowitz Neuromuscular Centre (DNC), at UCL ICH, (GOS-ICH), Great Ormond Street. Susanna is studying a MPharmacol at the University of Bath. Her work focuses on modelling the molecular and cellular mechanisms underlying skeletal muscle biology and regenerative processes in neuromuscular diseases. Susanna is motivated by GTAC’s mission to translate research into novel therapies for patients, and is keen to contribute to research that will help improve muscle function and health.
Email: s.arachchige@ucl.ac.uk
Alexandra Cioffi is a research technician at the Genetic Therapy Accelerator Centre (GTAC), IoN, working alongside Dr Schnekenberg on the functional characterisation, cell-based modelling, and subsequent therapeutic development for ITPR1-related disorders. After completing her MRes in Molecular and Cellular Biosciences at Imperial College London, she worked as a scientist in multiple research teams supporting allogeneic and autologous therapy platforms. Through joining GTAC, she continues her interest in the development of novel gene and cell therapies and aspires to advance research in this field.
Email: a.cioffi@ucl.ac.uk