The Next Frontier in Children’s Health
The Next Frontier in Children’s Health
Although recent decades have seen great advances in the prevention and treatment of childhood disease, from viral and bacterial infection to many forms of cancer, modern medicine still has no effective answer to the vast majority of genetic conditions that affect hundreds of thousands of children in the UK alone, and many more across the world.
This is set to change. Technological leaps in recent years have opened up a whole new frontier in medical discovery, and Cambridge is at the leading edge of this transformation. Our researchers are harnessing the power of rapid genome sequencing, big data and artificial intelligence, stem cell manipulation, and gene editing to develop new diagnostics and treatments for children suffering from genetic disorders.
The panel discussed the University’s research initiative in this field, which is a major collaboration involving multiple departments and institutes across the University’s School of Clinical Medicine as well as our clinical partners at Cambridge University Hospitals.
The panel also discussed how our research initiative is closely linked to the Cambridge Children’s Hospital – a partnership between the University and the NHS to create a pioneering new hospital that will fully integrate mental and physical healthcare, with genomic medicine at its heart.
The recording of this event can be viewed on our YouTube here.
Prof. Julian Rayner (Chair) (Trinity 1993)
Prof. Rayner is Professor of Cell Biology and Director of the Cambridge Institute for Medical Research (CIMR). CIMR is a world-leading centre that brings together fundamental and clinical scientists to understand how cells work, and how normal cellular function is disrupted in genetic and infectious disease. CIMR is collaborating with the University’s Department of Paediatrics to discover the molecular and cellular mechanisms of genetic disease in children, providing insights that are essential to the development of new diagnostics and treatments. Prof. Rayner is also strongly committed to public engagement with research, and is Director of Wellcome Connecting Science, which aims to enable everyone to explore genomics and its impact on research, health, and society.
Prof. David Rowitch (Clare 1984)
Prof. Rowitch is Head of the University’s Department of Paediatrics, academic lead for the new Cambridge Children’s Hospital, and holds a joint appointment in paediatrics and neurological surgery at the University of California, San Francisco. Prof. Rowitch is a clinician-scientist using genomic technologies to develop new diagnostics and treatments, including gene and cell therapies, for genetic neurological disorders in children, such as cerebral palsy and leukodystrophy. He led the first human clinical trial of stem cell transplantation for the fatal leukodystrophy, Pelizaeus-Merzbacher Disease. Prof. Rowitch sits on the US National Advisory Council for Children’s Health and Human Development (NACHHD) and is ‘Genomics Champion’ for the Academy of Royal Medical Colleges in the UK.
Dr Delphine Larrieu
Dr Larrieu is a Sir Henry Dale Fellow and Principal Investigator at the Cambridge Institute for Medical Research (CIMR). Dr Larrieu’s research group specialises in progerias, genetic conditions that cause severe premature ageing in children. Her research focuses on investigating the molecular mechanisms behind progerias, and on developing genetic screening technology to identify new therapeutic strategies for these diseases. Dr Larrieu is also Co-Founder of Adrestia Therapeutics, a spin-out company based on her scientific work, whose aim it is to develop new methods of restoring the biological balance disturbed by genetic conditions.
Prof. Evan Reid
Prof. Reid is Professor of Neurogenetics and Molecular Neurobiology as well as a Consultant in Medical Genetics at Cambridge’s Addenbrooke’s Hospital, where he runs a specialised neurogenetics clinic. His research group is based at the Cambridge Institute for Medical Research (CIMR) and is focused on hereditary spastic paraplegias. These are genetic forms of motor neuron degeneration that cause difficulties with walking independently, frequently starting in childhood and progressively worsening over time. Prof. Reid’s research encompasses the clinical features, genetics, and cell biology of hereditary spastic paraplegias, concentrating on understanding their molecular pathology with the aim of designing new treatments for these disorders.
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