Next-generation models and genetic therapies for rare neuromuscular diseases

Projektinformationen

MAGIC

ID Finanzhilfevereinbarung: 101080690

DOI

10.3030/101080690

EK-Unterschriftsdatum 28 April 2023

Startdatum 1 Juni 2023

Enddatum 31 Mai 2027

Finanziert unter

• Health

Gesamtkosten

€ 6 643 472,00

EU-Beitrag

€ 6 553 472,00

Koordiniert durch

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France

CORDIS bietet Links zu öffentlichen Ergebnissen und Veröffentlichungen von HORIZONT-Projekten.

Links zu Ergebnissen und Veröffentlichungen von RP7-Projekten sowie Links zu einigen Typen spezifischer Ergebnisse wie Datensätzen und Software werden dynamisch von OpenAIRE abgerufen.

Leistungen

Data Management Plan (1)

Data Management Plan

A Data Management Plan (DMP) following the HE guidelines will address the relevant aspects of making data FAIR, including what data the project will generate, whether and how it will be made accessible for verification and re-use, and how it will be curated and preserved. The DMP will also include details of the security measures to be taken in the context of the transfer of data from non-EU countries to prevent unauthorised access to personal data.

Websites, patent fillings, videos etc. (1)

Project website

Report on the design, set up and maintenance of the website

Documents, reports (4)

3D muscle generation

Protocol to generate advanced disease-specific 3D muscle models

Exploitation Plan (first release)

This plan is a strategic document indicating how the consortium establishes the basis for the intellectual property management and exploitation of key research outputs. It will include a preliminary analysis of analysis of both the research and market landscapes

Communication and Dissemination plan (first release)

Review and update (where necessary) of the original Communication and Dissemination Plan included in Annex 1

Perfusion, stimulation & miniaturisation of 3D muscles

Method to perfuse, stimulate and miniaturise 3D muscle models

Veröffentlichungen

Peer reviewed articles (7)

CRISPR-Cas9-mediated upregulation of utrophin ameliorates Duchenne muscular dystrophy

Autoren: Maëlle Ralu, Simon Guiraud, Sumitava Dastidar, Paola Galbiati, Emilia Sadaoui, Fetta Mazed, Fatima Amor, Anne de Cian, Isabelle Richard, Kamel Mamchaoui, Giuseppe Ronzitti, Francesco Saverio Tedesco, Mario Amendola
Veröffentlicht in: Molecular Therapy, Ausgabe 34, 2026, ISSN 1525-0016
Herausgeber: Elsevier BV
DOI: 10.1016/J.YMTHE.2026.03.025

Extensive striated muscle damage in a rat model of Duchenne muscular dystrophy with Dmd exons 10–17 duplication

Autoren: Jean-Daniel Masson, Valentina Taglietti, François Ruby, Hiroya Ono, Nadir Mouri, Alan Jorge, Laurent Guillaud, Laurent Tiret, Frederic Relaix
Veröffentlicht in: Skeletal Muscle, Ausgabe 15, 2025, ISSN 2044-5040
Herausgeber: Springer Science and Business Media LLC
DOI: 10.1186/S13395-025-00386-2

Low-cost, versatile, and highly reproducible microfabrication pipeline to generate 3D-printed customised cell culture devices with complex designs

Autoren: Cathleen Hagemann, Matthew C. D. Bailey, Eugenia Carraro, Ksenia S. Stankevich, Valentina Maria Lionello, Noreen Khokhar, Pacharaporn Suklai, Carmen Moreno-Gonzalez, Kelly O’Toole, George Konstantinou, Christina L. Dix, Sudeep Joshi, Eleonora Giagnorio, Mads S. Bergholt, Christopher D. Spicer, Albane Imbert, Francesco Saverio Tedesco, Andrea Serio
Veröffentlicht in: PLOS Biology, Ausgabe 22, 2024, ISSN 1545-7885
Herausgeber: Public Library of Science (PLoS)
DOI: 10.1371/journal.pbio.3002503

CD8+ T cell activation occurs 24 hours after AAV administration and is driven by muscle promoter specificity

Autoren: Lindsay Jeanpierre, Coralie Pecquet, Hanadi Saliba, Pauline Finard, Stéphane Terry, Gianni Tavella, Inès Guesmia, Sylvie Boutin, Bérangère Bertin, Sofia Benkhelifa-Ziyyat, Giuseppe Ronzitti, David-Alexandre Gross
Veröffentlicht in: JCI Insight, Ausgabe 11, 2026, ISSN 2379-3708
Herausgeber: American Society for Clinical Investigation
DOI: 10.1172/JCI.INSIGHT.202541

Forskolin treatment enhances muscle regeneration and shows therapeutic potential with limitations in Duchenne muscular dystrophy

Autoren: Andreea Iuliana Cojocaru, Kaouthar Kefi, Jean-Daniel Masson, Laurent Tiret, Frederic Relaix, Valentina Taglietti
Veröffentlicht in: Skeletal Muscle, Ausgabe 15, 2025, ISSN 2044-5040
Herausgeber: Springer Science and Business Media LLC
DOI: 10.1186/S13395-025-00381-7

Molecular Therapy

Autoren: Maëlle Ralu; Simon Guiraud; Sumitava Dastidar; Paola Galbiati; Emilia Sadaoui; Fetta Mazed; Fatima Amor; Anne de Cian; Isabelle Richard; Kamel Mamchaoui; Giuseppe Ronzitti; Francesco Saverio Tedesco; Mario Amendola
Veröffentlicht in: Molecular Therapy, Ausgabe 34, 2026, ISSN 1525-0016
Herausgeber: Elsevier
DOI: 10.1016/J.YMTHE.2026.03.025

Generation of a human induced pluripotent stem cell line (CRICKi021-A) from a patient with Ullrich congenital muscular dystrophy carrying a pathogenic mutation in the COL6A1 gene

Autoren: Yunsong Jiang, Liani G. Devito, Francesco Muntoni, Lyn Healy, Francesco Saverio Tedesco
Veröffentlicht in: Stem Cell Research, Ausgabe 83, 2025, ISSN 1873-5061
Herausgeber: Elsevier BV
DOI: 10.1016/j.scr.2024.103648