Objetivo Sudden cardiac death (SCD) is a leading cause of death in western countries: coronary artery disease is the major cause of SCD in older subjects while inherited arrhythmogenic diseases are the leading cause of SCD in younger individuals. After 25 years dedicated to research of the molecular bases of heritable arrhythmias, the PI of this proposal now intends to pioneer gene therapy for prevention of SCD: a virtually unexplored field. The development of molecular therapies for rhythm disturbances is a high risk effort however, if successful, it will be highly rewarding. The PI has envisioned an ambitious and comprehensive project to target two severe inherited arrhythmogenic diseases: dominant catecholaminergic polymorphic ventricular tachycardia (CPVT) and Long QT syndrome type 8 (LQT8). The availability of a clinically relevant model is critical to ensure clinical translation of results: the team will exploit an existing CPVT model and will engineer a knock-in pig to model LQT8. The PI and her team will investigate innovative strategies of gene-delivery, gene-silencing and gene-editing to the heart comparing efficacy of different constructs and promoters. The team will also carefully engineer novel gene-therapy approaches to avoid the development of regional inhomogeneity in protein expression that may facilitate proarrhythmic events. Such a comprehensive approach will provide a most valuable core of knowledge on the comparative efficacy of a broad range of molecular strategies on the electrical milieu of the heart. It is expected that these results will not only benefit CPVT and LQT8 but rather they will foster development of gene therapy for other inherited and acquired arrhythmias. Ámbito científico medical and health sciencesclinical medicinecardiologycardiovascular diseasescardiac arrhythmiamedical and health sciencesmedical biotechnologygenetic engineeringgene therapymedical and health sciencesclinical medicineobstetricsmedical and health sciencesbasic medicinephysiologymedical and health sciencesclinical medicineembryology Programa(s) H2020-EU.1.1. - EXCELLENT SCIENCE - European Research Council (ERC) Main Programme Tema(s) ERC-ADG-2014 - ERC Advanced Grant Convocatoria de propuestas ERC-2014-ADG Consulte otros proyectos de esta convocatoria Régimen de financiación ERC-ADG - Advanced Grant Institución de acogida UNIVERSITA DEGLI STUDI DI PAVIA Aportación neta de la UEn € 1 334 029,00 Dirección STRADA NUOVA 65 27100 Pavia Italia Ver en el mapa Región Nord-Ovest Lombardia Pavia 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 € 1 334 029,00 Beneficiarios (3) Ordenar alfabéticamente Ordenar por aportación neta de la UE Ampliar todo Contraer todo UNIVERSITA DEGLI STUDI DI PAVIA Italia Aportación neta de la UEn € 1 334 029,00 Dirección STRADA NUOVA 65 27100 Pavia Ver en el mapa Región Nord-Ovest Lombardia Pavia 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 € 1 334 029,00 AVANTEA SRL Italia Aportación neta de la UEn € 380 000,00 Dirección VIA CABRINI 12 26100 CREMONA Ver en el mapa Pyme Organización definida por ella misma como pequeña y mediana empresa (pyme) en el momento de la firma del acuerdo de subvención. Sí Región Nord-Ovest Lombardia Cremona Tipo de actividad Private for-profit entities (excluding 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 € 380 000,00 CENTRO NACIONAL DE INVESTIGACIONES CARDIOVASCULARES CARLOS III (F.S.P.) España Aportación neta de la UEn € 600 000,00 Dirección CALLE MELCHOR FERNANDEZ ALMAGRO 3 28029 Madrid Ver en el mapa Región Comunidad de Madrid Comunidad de Madrid Madrid 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 € 600 000,00