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Authors
Rossiaud, Lucille
Miagoux, Quentin
Benabides, Manon
Reiss, Océane
Jauze, Louisa
Jarrige, Margot
Polvèche, Hélène https://orcid.org/0000-0002-5601-3498
Malfatti, Edoardo
Laforêt, Pascal
Ronzitti, Giuseppe
Nissan, Xavier https://orcid.org/0000-0003-3374-6274
Hoch, Lucile https://orcid.org/0000-0002-7264-9031
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Funding
Funding for this research was provided by:
Agence Nationale de la Recherche (ANR-22-CE17-0031)
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Article History
Received: 11 October 2024
Revised: 6 March 2025
Accepted: 27 March 2025
First Online: 14 April 2025
Competing interests
: The authors declare no competing interests. GR is the author of patents related to the development of AAV gene therapy approaches for GSDIII.
: All methods were performed in accordance with the relevant guidelines and regulations. Human muscle biopsies were collected from patients and healthy donors after obtaining written informal consent, according to the French legislation and approved by the Comité de Protection des Personnes (approval number: Est IV DC-2012-1693). All animals were handled according to French and European guidelines on animal care and experimentation. All procedures on animals were approved by the local institutional ethical committee, Comité d’éthique de Genopole en expérimentation animale (approval number: CEEA - 051 [Evry, France]).
