CORDIS fornisce collegamenti ai risultati finali pubblici e alle pubblicazioni dei progetti ORIZZONTE.
I link ai risultati e alle pubblicazioni dei progetti del 7° PQ, così come i link ad alcuni tipi di risultati specifici come dataset e software, sono recuperati dinamicamente da .OpenAIRE .
Risultati finali
A Data Management Plan (DMP) will be issued on M6 as a live document that will be updated on a yearly basis concerning all project management data (e.g., including dissemination related data/contacts). It will be elaborated considering the previous open access rules and research data management. A detailed description of the knowledge and further exploitation activities will be provided within the DMP.
PEDR document will describe in detail the communication, dissemination and exploitation strategy in all its components: objectives of the plan, specific audiences to target, channels used to reach the different targets, actions to implement, dissemination of results, IP and exploitation tools.
Characterization and Quality control of iPSC lines and myogenic derivatives. I (si apre in una nuova finestra)A report containing a molecular characterization (expression of pluripotency markers, embryoid bodies formation, karyotypic stability) of healthy, Cas9 inducible and patients with the 5 starting NMD iPSC. This report will also contain a detailed molecular characterization of their myogenic derivatives (banking reports, expression of myogenic markers, myoblast proliferation, myotubes terminal differentiation).
Regulatory and ethics approval for blood sample collection (si apre in una nuova finestra)These documents will be submitted to, reviewed and validated by a French Ethical Committee (Comité de Protection des Personnes – CPP) to ensure a total compliance with French and European laws on biomedical research.
Quality Report (si apre in una nuova finestra)Quality assurance (QA) guidelines and procedures. A set of QA principles.To supervise the technical implementation of the project, ensuring not only efficient and timely implementation of tasks, but also that high quality standards are met, a Project management handbook and quality plan will be defined by Project Coordinator (PC) and the Project Technical Committee (PTC – composed by WP leaders) , including all relevant project procedures.
Characterization and Quality control of iPSC lines and myogenic derivatives. II (si apre in una nuova finestra)Updating - Characterization and Quality control of iPSC lines and myogenic derivatives. A report containing a molecular characterization (expression of pluripotency markers, embryoid bodies formation, karyotypic stability) of healthy, Cas9 inducible and patients with the 5 starting NMD iPSC. This report will also contain a detailed molecular characterization of their myogenic derivatives (banking reports, expression of myogenic markers, myoblast proliferation, myotubes terminal differentiation).
Report on communication and dissemination activities. I (si apre in una nuova finestra)A detailed list of communication and dissemination activities with assessment of their impact, degree of compliance with the plan, and a description of foreseen activities until the end of the project.
Consent form preparation involving patients (si apre in una nuova finestra)All regulatory documents required for sample collection, and in particular patient documents (consent and information leaflet), which will have been proofread by a committee of expert patients to ensure that they can be understood by the general public. These documents will ensure compliance with French and European laws on biomedical research. Patients will be involved in the writing and review of all regulatory documents required for sample collection, and in particular patient documents (consent and information leaflet) to ensure that they can be understood by the general public.
Creation of the External Advisory Board (EAB) led by the coordinator CECS in WP8. EAB composition includes key stakeholders such as Prof Volker Straub (Deputy Dean, Harold Macmillan Professor of Medicine and Professor of Neuromuscular Genetics at the Institute of Translational and Clinical Research at Newcastle University, Coordinator of the European consortium TREAT-NMD, and more importantly he is the new president of the World Muscle Society), Prof Nicolas Levy (APHM, expert in neuromuscular disorders and Chief Scientist Rare Diseases in the Pharmaceutical Group Servier), Dr Pascale Bomont (INMG, expert in the use of computational biology in NMDs and ERC grantee), Prof Laurent Servais (Professor of Paediatric Neuromuscular Diseases at MDUK Oxford Neuromuscular Center, University of Oxford), and Prof Hugues Bersini (Director of the AI Lab of the Université Libre de Bruxelles and co-founder of FARI (FARI | AI Institute for the Common Good, Brussels)).
Creation of The Patient Advisory Board (si apre in una nuova finestra)Patients from The Patient Advisory Board of the European Reference Network for Neuromuscular Diseases (ERN EuroNMD) could be implicated in the Patient Advisory group of the project DREAMS. AFM-Telethon will lead the creation of this Board and will proceed with the required contacts. The DREAMS Consortium will agree on the composition of the Board before formalising The Patient Advisory Board of DREAMS.
Project Website (si apre in una nuova finestra)Website of DREAMS´ project will be designed and fully operative by month 3 of the project. Development of project website includes: design, setup, and continuous updates throughout the project duration. It will contain general information, latest updates/news, links with social media channels, communication material and press releases/articles around the project, and related topics.
Pubblicazioni
Autori:
Celine Bruge, Nathalie Bourg, Emilie Pellier, Johana Tournois, Jerome Polentes, Manon Benabides, Noella Grossi, Anne Bigot, Anthony Brureau, Isabelle Richard, Xavier Nissan
Pubblicato in:
British Journal of Pharmacology, Numero 182, 2025, ISSN 0007-1188
Editore:
Wiley
DOI:
10.1111/BPH.70017
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