Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy.

"supplementary figure s11 data availability data will be made available on request."

"Declaration of competing interest D.G. is a cofounder of AaviGen GmbH. F.M. works at Spark Therapeutics. D.G., J.E.A., T.V., M.K.C. and W.T. are inventors on patent applications related to this work. All other authors declare that they have no competing interests. The AAV plasmid bearing the GAA expression cassette including a full map can be obtained via an MTA from the Vrije Universiteit Brussel. The AAVMYO3 helper plasmid including a full map can easily be obtained by academic institutions via a standard non-commercial MTA from Heidelberg University Hospital."

"We thank J. Barrero, L. Hernández, M. Moya, Á. Vázquez, L. Noya and S. Frankhauser for technical assistance. We kindly acknowledge the support provided by the MYOCURE project to F.B., M.K.C., T.V., D.G. and F.M. MYOCURE has received funding from the European Union’s Horizon 2020 research and innovation programme under grant agreement No. 667751. F.B. received support from MCIN/AEI/10.13039/501100011033 (PID2020-113864RB-100), from 10.13039/501100002809Generalitat de Catalunya (2021 SGR 01012, ICREA Academia Award to F.B.) and from CIBER-Consorcio Centro de Investigación Biomédica en Red- (CB07/08/0037), Instituto de Salud Carlos III, Ministerio de Ciencia e Innovación. V.S. was recipient of a predoctoral fellowship (Grant FPU2014-FPU14/05268) funded by MCIN/AEI/10.13039/501100011033 and “ERDF A way of making Europe”. T.V. and M.K.C. received grant support from the Vrije Universiteit Brussel Industrieel Onderzoeksfonds (IOF) Groups of Expertise in Advanced Research (GEAR) and Strategic Research Project (SRP Grower), F.W.O. (Fonds Wetenschappelijk Onderzoek Vlaanderen), 10.13039/501100006282Koning Boudewijnstichting (Fonds Walter Pyleman, Cremers-Opdebeeck, Richard Depasse). Sergio Muñoz: Conceptualization, Formal analysis, Investigation, Visualization, Writing – original draft, Writing – review & editing. Joan Bertolin: Conceptualization, Formal analysis, Investigation, Visualization, Writing – original draft. Veronica Jimenez: Conceptualization, Investigation, Supervision, Writing – original draft, Writing – review & editing. Maria Luisa Jaén: Data curation. Miquel Garcia: Data curation. Anna Pujol: Methodology, Resources. Laia Vilà: Data curation. Victor Sacristan: Data curation. Elena Barbon: Methodology, Resources. Giuseppe Ronzitti: Methodology, Resources. Jihad El Andari: Methodology, Resources. Warut Tulalamba: Methodology, Resources. Quang Hong Pham: Methodology, Resources. Jesus Ruberte: Data curation. Thierry VandenDriessche: Funding acquisition, Writing – review & editing. Marinee K. Chuah: Funding acquisition, Writing – review & editing. Dirk Grimm: Funding acquisition, Writing – review & editing. Federico Mingozzi: Conceptualization, Funding acquisition, Writing – review & editing. Fatima Bosch: Conceptualization, Funding acquisition, Investigation, Supervision, Writing – original draft, Writing – review & editing."