Objectif 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. Champ scientifique 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 Programme(s) H2020-EU.1.1. - EXCELLENT SCIENCE - European Research Council (ERC) Main Programme Thème(s) ERC-ADG-2014 - ERC Advanced Grant Appel à propositions ERC-2014-ADG Voir d’autres projets de cet appel Régime de financement ERC-ADG - Advanced Grant Institution d’accueil UNIVERSITA DEGLI STUDI DI PAVIA Contribution nette de l'UE € 1 334 029,00 Adresse STRADA NUOVA 65 27100 Pavia Italie Voir sur la carte Région Nord-Ovest Lombardia Pavia Type d’activité Higher or Secondary Education Establishments Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 1 334 029,00 Bénéficiaires (3) Trier par ordre alphabétique Trier par contribution nette de l'UE Tout développer Tout réduire UNIVERSITA DEGLI STUDI DI PAVIA Italie Contribution nette de l'UE € 1 334 029,00 Adresse STRADA NUOVA 65 27100 Pavia Voir sur la carte Région Nord-Ovest Lombardia Pavia Type d’activité Higher or Secondary Education Establishments Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 1 334 029,00 AVANTEA SRL Italie Contribution nette de l'UE € 380 000,00 Adresse VIA CABRINI 12 26100 CREMONA Voir sur la carte PME L’entreprise s’est définie comme une PME (petite et moyenne entreprise) au moment de la signature de la convention de subvention. Oui Région Nord-Ovest Lombardia Cremona Type d’activité Private for-profit entities (excluding Higher or Secondary Education Establishments) Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 380 000,00 CENTRO NACIONAL DE INVESTIGACIONES CARDIOVASCULARES CARLOS III (F.S.P.) Espagne Contribution nette de l'UE € 600 000,00 Adresse CALLE MELCHOR FERNANDEZ ALMAGRO 3 28029 Madrid Voir sur la carte Région Comunidad de Madrid Comunidad de Madrid Madrid Type d’activité Research Organisations Liens Contacter l’organisation Opens in new window Site web Opens in new window Participation aux programmes de R&I de l'UE Opens in new window Réseau de collaboration HORIZON Opens in new window Coût total € 600 000,00