Next-generation models and genetic therapies for rare neuromuscular diseases

Información del proyecto

MAGIC

Identificador del acuerdo de subvención: 101080690

DOI

10.3030/101080690

Fecha de la firma de la CE 28 Abril 2023

Fecha de inicio 1 Junio 2023

Fecha de finalización 31 Mayo 2027

Financiado con arreglo a

• Health

Coste total

€ 6 643 472,00

Aportación de la UE

€ 6 553 472,00

Coordinado por

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France

CORDIS proporciona enlaces a los documentos públicos y las publicaciones de los proyectos de los programas marco HORIZONTE.

Los enlaces a los documentos y las publicaciones de los proyectos del Séptimo Programa Marco, así como los enlaces a algunos tipos de resultados específicos, como conjuntos de datos y «software», se obtienen dinámicamente de OpenAIRE .

Resultado final

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

Publicaciones

Peer reviewed articles (7)

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

Autores: 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
Publicado en: Molecular Therapy, Edición 34, 2026, ISSN 1525-0016
Editor: 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

Autores: Jean-Daniel Masson, Valentina Taglietti, François Ruby, Hiroya Ono, Nadir Mouri, Alan Jorge, Laurent Guillaud, Laurent Tiret, Frederic Relaix
Publicado en: Skeletal Muscle, Edición 15, 2025, ISSN 2044-5040
Editor: 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

Autores: 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
Publicado en: PLOS Biology, Edición 22, 2024, ISSN 1545-7885
Editor: 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

Autores: 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
Publicado en: JCI Insight, Edición 11, 2026, ISSN 2379-3708
Editor: 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

Autores: Andreea Iuliana Cojocaru, Kaouthar Kefi, Jean-Daniel Masson, Laurent Tiret, Frederic Relaix, Valentina Taglietti
Publicado en: Skeletal Muscle, Edición 15, 2025, ISSN 2044-5040
Editor: Springer Science and Business Media LLC
DOI: 10.1186/S13395-025-00381-7

Molecular Therapy

Autores: 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
Publicado en: Molecular Therapy, Edición 34, 2026, ISSN 1525-0016
Editor: 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

Autores: Yunsong Jiang, Liani G. Devito, Francesco Muntoni, Lyn Healy, Francesco Saverio Tedesco
Publicado en: Stem Cell Research, Edición 83, 2025, ISSN 1873-5061
Editor: Elsevier BV
DOI: 10.1016/j.scr.2024.103648