Next-generation models and genetic therapies for rare neuromuscular diseases

Informacje na temat projektu

MAGIC

Identyfikator umowy o grant: 101080690

DOI

10.3030/101080690

Data podpisania przez KE 28 Kwietnia 2023

Data rozpoczęcia 1 Czerwca 2023

Data zakończenia 31 Maja 2027

Finansowanie w ramach

• Health

Koszt całkowity

€ 6 643 472,00

Wkład UE

€ 6 553 472,00

Koordynowany przez

INSTITUT NATIONAL DE LA SANTE ET DE LA RECHERCHE MEDICALE
France

CORDIS oferuje możliwość skorzystania z odnośników do publicznie dostępnych publikacji i rezultatów projektów realizowanych w ramach programów ramowych HORYZONT.

Odnośniki do rezultatów i publikacji związanych z poszczególnymi projektami 7PR, a także odnośniki do niektórych konkretnych kategorii wyników, takich jak zbiory danych i oprogramowanie, są dynamicznie pobierane z systemu OpenAIRE .

Rezultaty

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

Publikacje

Peer reviewed articles (7)

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

Autorzy: 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
Opublikowane w: Molecular Therapy, Numer 34, 2026, ISSN 1525-0016
Wydawca: 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

Autorzy: Jean-Daniel Masson, Valentina Taglietti, François Ruby, Hiroya Ono, Nadir Mouri, Alan Jorge, Laurent Guillaud, Laurent Tiret, Frederic Relaix
Opublikowane w: Skeletal Muscle, Numer 15, 2025, ISSN 2044-5040
Wydawca: 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

Autorzy: 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
Opublikowane w: PLOS Biology, Numer 22, 2024, ISSN 1545-7885
Wydawca: 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

Autorzy: 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
Opublikowane w: JCI Insight, Numer 11, 2026, ISSN 2379-3708
Wydawca: 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

Autorzy: Andreea Iuliana Cojocaru, Kaouthar Kefi, Jean-Daniel Masson, Laurent Tiret, Frederic Relaix, Valentina Taglietti
Opublikowane w: Skeletal Muscle, Numer 15, 2025, ISSN 2044-5040
Wydawca: Springer Science and Business Media LLC
DOI: 10.1186/S13395-025-00381-7

Molecular Therapy

Autorzy: 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
Opublikowane w: Molecular Therapy, Numer 34, 2026, ISSN 1525-0016
Wydawca: 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

Autorzy: Yunsong Jiang, Liani G. Devito, Francesco Muntoni, Lyn Healy, Francesco Saverio Tedesco
Opublikowane w: Stem Cell Research, Numer 83, 2025, ISSN 1873-5061
Wydawca: Elsevier BV
DOI: 10.1016/j.scr.2024.103648