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UCL Queen Square Institute of Neurology

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This Centre is able to spearhead activities in this rapidly evolving area and play a major role in translational neuroscience in UCL.

The Genetic Therapy Accelerator Centre (GTAC) is led by Professor Francesco Muntoni, an Internationally recognised Paediatric Neurologist with dual appointments at UCL QS Institute of Neurology. He is director of the Dubowitz Neuromuscular Centre, at the UCL GOS Institute of Child Health and Great Ormond Street Hospital for Children. The foundation of the centre builds on his pioneering work in the first genetic therapies for neuromuscular disorders and the vision to apply this knowledge and experience to develop the next generation of genetic therapies for a range of neurological disorders. The revolution in genomics has led to an explosion of potential genetic targets as novel therapeutic targets. The activities of the Centre is highly complementary with established expertise and research activities in both Institutes, including the Dementia Research Institute, the Sigrid Rausing Neurogenetics Therapies Programme and two NIHR Biomedical Research Centres translational research programmes (UCLH and GOSH).

In the last five years a number of extremely effective RNA and adeno-associated viral vectors (AAV) therapies have dramatically improved the outcomes for individuals affected by a range of neurological disorders, mostly affecting children. The focus of the centre is to harness the learning derived from these recent successes, mechanistic insight discovery, to early and late translational research and eventually regulatory approval, and apply it to a wider group of neurodegenerative disorders.  The centre combines laboratory and clinical expertise focused on neurological, neuromuscular and neurodegenerative disorders, their mechanisms of disease and cutting-edge RNA and AAV gene therapy expertise, and foster cross-institutional collaborations between IoN QS and GOS ICH. Professor Muntoni, along with Dr Ng and Dr Aguti lead the research programmes in translational AAV and RNA therapeutics. Centre staff provide expert advice and support to other departments at IoN QS and GOS ICH on translational genetic therapy developments from proof of concept, preclinical studies of efficacy and safety towards clinical translation.

The Centre develops links and synergies with other related activities including the Sigrid Rausing Trust Initiative, Dementia Research Institute, LMIC International Centre for Neuromuscular Diseases, Neurogenetics Therapies Programme, the two Biomedical Research Centres at IoNQS and GOSICH, and the genetic therapy Initiatives at the Zayed Centre for Research into Rare Disease in children.