A faulty genetic instruction during brain development can derail a child’s ability to learn, speak, or control their movements, often for life. These neurodevelopmental disorders—including epilepsy, intellectual disability, autism, and schizophrenia—are notoriously difficult to treat, partly because scientists still do not understand how specific mutations and environmental factors actually disrupt the brain’s long, precisely timed construction program. The King’s College London Centre will combine studies in human patients and animal models to trace exactly how these genetic errors cause the brain to develop abnormally. This is fundamental science: the immediate goal is not a new drug or diagnostic test, but a mechanistic understanding of where and when development goes wrong. Past discoveries in fundamental developmental biology have led to breakthroughs in treating conditions once considered untreatable. If this work succeeds, it could eventually reveal molecular targets for therapies that intervene early—before disability becomes entrenched—and help dissolve the current gap between laboratory research and clinical care.
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Neurodevelopmental disorders are a group of disorders in which the development of the brain is disturbed. This can manifest as neuropsychiatric problems or impaired motor function, learning, language or non-verbal communication. These disorders include epilepsy, intellectual disability, autism, and even other diseases with a relatively late onset, such as schizophrenia. These disorders are very difficult to treat and often cause a life-long disability. The development of the brain is a highly orchestrated process, controlled by genetic information under very clear influence from the environment. In humans, brain development prolongs from early in life to adolescence, when many connections between neurons, the cells of the brain, are actively remodelled. Any deviation from this very protracted program can result in neurodevelopmental disorders and, depending on specific timing, might lead to distinct pathology later in life. Neurodevelopmental disorders have a prominent genetic basis, although this does not necessarily mean that they are hereditary. For instance, many patients carry de novo mutations - an alteration in a gene that is present for the first time in one family member as a result of a mutation in a germ cell (egg or sperm) of one of the parents or in the fertilized egg itself. Recent advances in human genetics have identified specific mutations with these disorders. Unfortunately, we still do not understand how these mutations and concomitant environmental insults cause neurodevelopmental disorders. Our vision is to build a world-class Centre at King's College London with research programmes that will transform our understanding of the origin of neurodevelopmental disorders. To this end, we will investigate the biological mechanisms underlying these disorders in human patients and animal models. We will also train and nurture the next generation of scientific leaders in the field, equipped to dissolve the boundaries that exist between basic research and clinical disciplines. In the long term, we aim to translate the new knowledge into clinical advances that change the lives of affected individuals and their families, in collaboration with industrial partners, other research centres and patient associations.
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