Obiettivo Mitochondria are the major source of ATP, synthesized by the mitochondrial respiratory chain (MRC) through the process of oxidative phosphorylation. ATP deficiency leads to cellular dysfunction and ultimately death. In mammals, 13 mitochondrial DNA (mtDNA)-encoded subunits interact with over 70 nuclear-encoded subunits to form four of the five MRC complexes. Many additional factors are essential for the regulation of MRC activity, and the maintenance and expression of mtDNA. As a result, genetic defects affecting either genome can compromise ATP synthesis and cause human disease. There is no effective treatment for mitochondrial disorders. Major hurdles to this achievement include (i) the still incomplete molecular definition of mitochondrial disease; (ii) the poorly understood function of many disease gene products, (iii) the difficulty to rationally manipulate the complex biochemical and genetic systems underpinning mitochondrial bioenergetics.The ultimate scope of MitCare is to develop effective therapy in mitochondrial medicine. MitCare will implement three Workpackages (WP). WP1 will test the effects of pharmacological stimulation of mitochondrial biogenesis in mouse disease models. WP2 will test the effects of Adeno-Associated Viral (AAV)- and lentiviral-mediated delivery of therapeutic genes, in mouse models and human mutant cells, respectively. Since the clinical features of human disorders often fail to be faithfully replicated in mice, WP3 will implement the creation of a mitochondrial disease model in the pig, whose proximity to humans is much closer than mice. The Surf1 gene, encoding an assembly factor of complex IV, will be disrupted by zincfinger nuclease technology in swine fibroblasts, which will then serve to clone a Surf1 knockout pig by somatic cell nuclear transfer. “Mitopigs” will be used to test the direct transferability of experimental treatments from suitable mammalian models to the clinics. Campo scientifico natural sciencesbiological sciencesgeneticsDNAnatural sciencesbiological scienceszoologymammalogynatural sciencesbiological sciencesgeneticsgenomes Programma(i) FP7-IDEAS-ERC - Specific programme: "Ideas" implementing the Seventh Framework Programme of the European Community for research, technological development and demonstration activities (2007 to 2013) Argomento(i) ERC-AG-LS4 - ERC Advanced Grant - Physiology, Pathophysiology and Endocrinology Invito a presentare proposte ERC-2012-ADG_20120314 Vedi altri progetti per questo bando Meccanismo di finanziamento ERC-AG - ERC Advanced Grant Istituzione ospitante THE CHANCELLOR MASTERS AND SCHOLARS OF THE UNIVERSITY OF CAMBRIDGE Contributo UE € 2 004 284,12 Indirizzo TRINITY LANE THE OLD SCHOOLS CB2 1TN Cambridge Regno Unito Mostra sulla mappa Regione East of England East Anglia Cambridgeshire CC Tipo di attività Higher or Secondary Education Establishments Ricercatore principale Massimo Zeviani (Prof.) Contatto amministrativo Renata Schaeffer Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato Beneficiari (4) Classifica in ordine alfabetico Classifica per Contributo UE Espandi tutto Riduci tutto THE CHANCELLOR MASTERS AND SCHOLARS OF THE UNIVERSITY OF CAMBRIDGE Regno Unito Contributo UE € 2 004 284,12 Indirizzo TRINITY LANE THE OLD SCHOOLS CB2 1TN Cambridge Mostra sulla mappa Regione East of England East Anglia Cambridgeshire CC Tipo di attività Higher or Secondary Education Establishments Ricercatore principale Massimo Zeviani (Prof.) Contatto amministrativo Renata Schaeffer Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato AVANTEA SRL Italia Contributo UE € 401 000,01 Indirizzo VIA CABRINI 12 26100 CREMONA Mostra sulla mappa Regione Nord-Ovest Lombardia Cremona Tipo di attività Private for-profit entities (excluding Higher or Secondary Education Establishments) Contatto amministrativo Giovanna Lazzari (Dr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato FONDAZIONE IRCCS ISTITUTO NEUROLOGICO CARLO BESTA Italia Contributo UE € 94 715,87 Indirizzo VIA GIOVANNI CELORIA 11 20133 Milano Mostra sulla mappa Regione Nord-Ovest Lombardia Milano Tipo di attività Research Organisations Contatto amministrativo Alberto Guglielmo (Mr.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato MEDICAL RESEARCH COUNCIL Partecipazione conclusa Regno Unito Contributo UE Nessun dato Indirizzo 20 Park Crescent W1B 1AL LONDON Mostra sulla mappa Tipo di attività Public bodies (excluding Research Organisations and Secondary or Higher Education Establishments) Contatto amministrativo Irina James (Mrs.) Collegamenti Contatta l’organizzazione Opens in new window Sito web Opens in new window Costo totale Nessun dato
