Obiettivo 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. Campo scientifico 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 Programma(i) H2020-EU.1.1. - EXCELLENT SCIENCE - European Research Council (ERC) Main Programme Argomento(i) ERC-ADG-2014 - ERC Advanced Grant Invito a presentare proposte ERC-2014-ADG Vedi altri progetti per questo bando Meccanismo di finanziamento ERC-ADG - Advanced Grant Istituzione ospitante UNIVERSITA DEGLI STUDI DI PAVIA Contribution nette de l'UE € 1 334 029,00 Indirizzo STRADA NUOVA 65 27100 Pavia Italia Mostra sulla mappa Regione Nord-Ovest Lombardia Pavia Tipo di attività Higher or Secondary Education Establishments Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 1 334 029,00 Beneficiari (3) Classifica in ordine alfabetico Classifica per Contributo netto dell'UE Espandi tutto Riduci tutto UNIVERSITA DEGLI STUDI DI PAVIA Italia Contribution nette de l'UE € 1 334 029,00 Indirizzo STRADA NUOVA 65 27100 Pavia Mostra sulla mappa Regione Nord-Ovest Lombardia Pavia Tipo di attività Higher or Secondary Education Establishments Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 1 334 029,00 AVANTEA SRL Italia Contribution nette de l'UE € 380 000,00 Indirizzo VIA CABRINI 12 26100 CREMONA Mostra sulla mappa PMI L’organizzazione si è definita una PMI (piccola e media impresa) al momento della firma dell’accordo di sovvenzione. Sì Regione Nord-Ovest Lombardia Cremona Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 380 000,00 CENTRO NACIONAL DE INVESTIGACIONES CARDIOVASCULARES CARLOS III (F.S.P.) Spagna Contribution nette de l'UE € 600 000,00 Indirizzo CALLE MELCHOR FERNANDEZ ALMAGRO 3 28029 Madrid Mostra sulla mappa Regione Comunidad de Madrid Comunidad de Madrid Madrid Tipo di attività Research Organisations Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Partecipazione a programmi di R&I dell'UE Opens in new window Rete di collaborazione HORIZON Opens in new window Costo totale € 600 000,00