Objetivo Skeletal muscle is the most abundant human tissue and contains mainly post-mitotic nuclei. It also expresses the largest gene known in nature – dystrophin – whose mutations cause Duchenne muscular dystrophy, the most frequent and incurable childhood muscle disorder. These characteristics create hurdles that negatively impact on the development of therapies for muscle diseases, ranging from acute tissue loss to chronic neuromuscular disorders. Moreover, a lack of humanised models of muscle regeneration delays the understanding of its regenerative dynamics.My work has pioneered the use of human induced pluripotent stem (iPS) cells to generate genetically corrected myogenic cells for the autologous cell therapy of muscular dystrophies. Here I propose to exploit this technology together with biocompatible materials to develop three dimensional, iPS cell-derived, patient-specific artificial muscles. These bioengineered skeletal muscles will provide a model to study human muscle regeneration and a platform for tissue engineering and therapy development for severe muscle diseases.The project will be developed in two phases. First we will develop the iPS cell-derived muscle in vitro, introducing cell types and stimuli necessary to obtain a functional tissue. In the second phase we will exploit the muscle “organoids” for regenerative medicine and drug development. Specifically, we will investigate the artificial muscle potential for tissue replacement in vivo and then model different muscular dystrophies in vitro to screen drugs with therapeutic relevance. Finally, we will combine the tools and knowledge developed in the two aforementioned areas into a novel platform to optimise skeletal muscle gene and cell therapies. This project will bring together tissue engineering, drug development and cell therapy under the same translational technology, advancing the understanding of pathogenesis and the development of therapies for muscle diseases. Ámbito científico medical and health sciencesbasic medicinepharmacology and pharmacydrug discoverymedical and health sciencesmedical biotechnologytissue engineeringmedical and health sciencesbasic medicineneurologymuscular dystrophiesduchenne muscular dystrophynatural sciencesbiological sciencesgeneticsmutationmedical and health sciencesmedical biotechnologycells technologies Palabras clave Disease modeling and tissue engineering Programa(s) H2020-EU.1.1. - EXCELLENT SCIENCE - European Research Council (ERC) Main Programme Tema(s) ERC-2017-STG - ERC Starting Grant Convocatoria de propuestas ERC-2017-STG Consulte otros proyectos de esta convocatoria Régimen de financiación ERC-STG - Starting Grant Institución de acogida UNIVERSITY COLLEGE LONDON Aportación neta de la UEn € 1 089 738,00 Dirección GOWER STREET WC1E 6BT London Reino Unido Ver en el mapa Región London Inner London — West Camden and City of London Tipo de actividad Higher or Secondary Education Establishments Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Participación en los programas de I+D de la UE Opens in new window Red de colaboración de HORIZON Opens in new window Coste total € 1 499 738,00 Beneficiarios (2) Ordenar alfabéticamente Ordenar por aportación neta de la UE Ampliar todo Contraer todo UNIVERSITY COLLEGE LONDON Reino Unido Aportación neta de la UEn € 1 089 738,00 Dirección GOWER STREET WC1E 6BT London Ver en el mapa Región London Inner London — West Camden and City of London Tipo de actividad Higher or Secondary Education Establishments Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Participación en los programas de I+D de la UE Opens in new window Red de colaboración de HORIZON Opens in new window Coste total € 1 499 738,00 Tercero Entidad jurídica distinta de un subcontratista que está afiliada o vinculada jurídicamente con un participante. Dicha entidad lleva a cabo su trabajo en las condiciones establecidas en el acuerdo de subvención, proporciona bienes o presta servicios para la acción, pero no firma dicho acuerdo. El tercero acata las normas aplicables a su participante en virtud del acuerdo de subvención en lo que respecta a la subvencionalidad de los costes y el control de los gastos. THE FRANCIS CRICK INSTITUTE LIMITED Reino Unido Aportación neta de la UEn € 410 000,00 Dirección 1 MIDLAND ROAD NW1 1AT London Ver en el mapa Región London Inner London — West Camden and City of London Tipo de actividad Research Organisations Enlaces Contactar con la organización Opens in new window Sitio web Opens in new window Participación en los programas de I+D de la UE Opens in new window Red de colaboración de HORIZON Opens in new window Coste total € 410 000,00